MX2023006169A - Terapia genica para trastornos neurodegenerativos. - Google Patents
Terapia genica para trastornos neurodegenerativos.Info
- Publication number
- MX2023006169A MX2023006169A MX2023006169A MX2023006169A MX2023006169A MX 2023006169 A MX2023006169 A MX 2023006169A MX 2023006169 A MX2023006169 A MX 2023006169A MX 2023006169 A MX2023006169 A MX 2023006169A MX 2023006169 A MX2023006169 A MX 2023006169A
- Authority
- MX
- Mexico
- Prior art keywords
- spinal
- gene therapy
- disclosed
- neurodegenerative disorders
- lateral sclerosis
- Prior art date
Links
- 238000001415 gene therapy Methods 0.000 title 1
- 208000015122 neurodegenerative disease Diseases 0.000 title 1
- 208000005264 motor neuron disease Diseases 0.000 abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 201000010901 lateral sclerosis Diseases 0.000 abstract 2
- 230000007659 motor function Effects 0.000 abstract 2
- 208000029402 Bulbospinal muscular atrophy Diseases 0.000 abstract 1
- 206010008025 Cerebellar ataxia Diseases 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 208000027747 Kennedy disease Diseases 0.000 abstract 1
- 208000032319 Primary lateral sclerosis Diseases 0.000 abstract 1
- 206010046298 Upper motor neurone lesion Diseases 0.000 abstract 1
- 208000027418 Wounds and injury Diseases 0.000 abstract 1
- 210000004556 brain Anatomy 0.000 abstract 1
- 230000006378 damage Effects 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 208000014674 injury Diseases 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 210000000278 spinal cord Anatomy 0.000 abstract 1
- 208000020431 spinal cord injury Diseases 0.000 abstract 1
- 208000002320 spinal muscular atrophy Diseases 0.000 abstract 1
- 230000000472 traumatic effect Effects 0.000 abstract 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/861—Adenoviral vectors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Marine Sciences & Fisheries (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Toxicology (AREA)
- Pain & Pain Management (AREA)
- Neurosurgery (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Se describen composiciones y métodos para tratar trastornos que afectan a la función motora, tal como función motora afectada por enfermedad o lesión del cerebro y/o la médula espinal. También se describe un vector de virus adeno asociado para tratar trastornos de las neuronas motoras tales como atrofia muscular espinal, esclerosis lateral amiotrófica, atrofia muscular bulbar espinal, ataxia cerebelar espinal, esclerosis lateral primaria y lesión traumática de la médula espinal.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US17498209P | 2009-05-02 | 2009-05-02 | |
US26805909P | 2009-06-08 | 2009-06-08 |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2023006169A true MX2023006169A (es) | 2023-06-09 |
Family
ID=43050324
Family Applications (4)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2016010101A MX356669B (es) | 2009-05-02 | 2010-04-27 | Terapia genica para trastornos neurodegenerativos. |
MX2011011594A MX341073B (es) | 2009-05-02 | 2010-04-27 | Terapia genica para trastornos neurodegenerativos. |
MX2023006169A MX2023006169A (es) | 2009-05-02 | 2011-11-01 | Terapia genica para trastornos neurodegenerativos. |
MX2018006955A MX2018006955A (es) | 2009-05-02 | 2011-11-01 | Terapia genica para trastornos neurodegenerativos. |
Family Applications Before (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2016010101A MX356669B (es) | 2009-05-02 | 2010-04-27 | Terapia genica para trastornos neurodegenerativos. |
MX2011011594A MX341073B (es) | 2009-05-02 | 2010-04-27 | Terapia genica para trastornos neurodegenerativos. |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2018006955A MX2018006955A (es) | 2009-05-02 | 2011-11-01 | Terapia genica para trastornos neurodegenerativos. |
Country Status (22)
Country | Link |
---|---|
US (7) | US20130287736A1 (es) |
EP (4) | EP2424991B1 (es) |
JP (3) | JP5879256B2 (es) |
KR (2) | KR101835490B1 (es) |
CN (2) | CN102459611B (es) |
BR (1) | BRPI1010868A2 (es) |
CA (1) | CA2759801C (es) |
CY (1) | CY1120982T1 (es) |
DK (2) | DK2424991T3 (es) |
ES (2) | ES2686504T3 (es) |
HR (2) | HRP20212024T1 (es) |
HU (2) | HUE057606T2 (es) |
IL (2) | IL216094A0 (es) |
LT (2) | LT3421603T (es) |
MX (4) | MX356669B (es) |
PL (2) | PL2424991T3 (es) |
PT (2) | PT3421603T (es) |
RS (1) | RS62779B1 (es) |
RU (2) | RU2603740C2 (es) |
SG (3) | SG10201404528SA (es) |
SI (2) | SI2424991T1 (es) |
WO (1) | WO2010129021A1 (es) |
Families Citing this family (80)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
HUE057606T2 (hu) | 2009-05-02 | 2022-05-28 | Genzyme Corp | Génterápia neurodegeneratív rendellenességekre |
WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
EP3444346B1 (en) | 2010-04-23 | 2022-07-27 | University of Massachusetts | Aav-based treatment of cholesterol-related disorders |
EP2826860B1 (en) * | 2010-04-23 | 2018-08-22 | University of Massachusetts | CNS targeting AAV vectors and methods of use thereof |
CN103491974A (zh) * | 2010-11-16 | 2014-01-01 | 神经机能生命科学公司 | 用于提高中性溶酶的表达与活性的方法 |
GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
EP2700399B1 (en) * | 2011-04-18 | 2017-05-31 | National Center of Neurology and Psychiatry | Drug delivery particles and method for producing same |
US9226976B2 (en) | 2011-04-21 | 2016-01-05 | University Of Massachusetts | RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
WO2013190059A1 (en) * | 2012-06-21 | 2013-12-27 | Association Institut De Myologie | Widespread gene delivery of gene therapy vectors |
ES2684222T3 (es) | 2012-08-01 | 2018-10-01 | Nationwide Children's Hospital | Administración intratecal de virus adenoasociado 9 recombinante |
CN103638604B (zh) * | 2012-12-31 | 2016-05-04 | 深圳先进技术研究院 | 一种助行*** |
WO2014169087A2 (en) * | 2013-04-10 | 2014-10-16 | The Trustees Of Columbia University In The City Of New York | Treatment of proximal spinal muscular atrophy |
US10821154B2 (en) | 2013-05-01 | 2020-11-03 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
US11408899B2 (en) | 2013-07-12 | 2022-08-09 | The Children's Hospital Of Philadelphia | AAV vector and assay for anti-AAV (adeno-associated virus) neutralizing antibodies |
US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
EP3119406B1 (en) | 2014-03-18 | 2021-01-20 | Carmel-Haifa University Economic Corporation Ltd | Methods for improving cognitive function via modulation of quinone reductase 2 |
US10280418B2 (en) | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
EP2933335A1 (en) * | 2014-04-18 | 2015-10-21 | Genethon | A method of treating peripheral neuropathies and motor neuron diseases |
WO2015164723A1 (en) | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
US10711270B2 (en) | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
RU2020140209A (ru) | 2014-10-21 | 2021-01-25 | Юниверсити Оф Массачусетс | Варианты рекомбинантных aav и их применения |
CA2966620A1 (en) | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
LT3237618T (lt) * | 2014-12-24 | 2019-07-10 | Uniqure Ip B.V. | Rnri sukeltas hantingtino geno slopinimas |
WO2016131009A1 (en) | 2015-02-13 | 2016-08-18 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
WO2016172008A1 (en) | 2015-04-24 | 2016-10-27 | University Of Massachusetts | Modified aav constructions and uses thereof |
AU2016315699B2 (en) * | 2015-08-31 | 2021-09-09 | The Trustees Of The University Of Pennsylvania | AAV-EPO for treating companion animals |
PT3356390T (pt) | 2015-09-28 | 2021-04-21 | Univ Florida | Métodos e composições para vetores virais que se evadem a anticorpos |
CA3002980A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
CA3002982A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
US11826433B2 (en) | 2016-02-02 | 2023-11-28 | University Of Massachusetts | Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system |
US11060088B2 (en) | 2016-02-12 | 2021-07-13 | University Of Massachusetts | Anti-angiogenic miRNA therapeutics for inhibiting corneal neovascularization |
WO2017151884A1 (en) * | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
WO2017176929A1 (en) | 2016-04-05 | 2017-10-12 | University Of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
JP6671664B2 (ja) * | 2016-05-24 | 2020-03-25 | 学校法人東京女子医科大学 | Smnタンパク質の核内構造体の発現解析方法 |
US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
CN110191956A (zh) * | 2016-11-07 | 2019-08-30 | 麦考瑞大学 | 蛋白质积累的调节及为其应用 |
JOP20190200A1 (ar) * | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
EP3622073A4 (en) | 2017-05-09 | 2021-01-06 | University of Massachusetts | METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
EP3668891B1 (en) * | 2017-08-16 | 2023-07-26 | Lgv1 S.R.L. | Vtft isoform of a bpifb4 protein for use in neuronal deseases and injuries |
EP3684937A4 (en) | 2017-09-22 | 2021-06-02 | University of Massachusetts | DUAL EXPRESSION VECTORS OF SOD1 AND THEIR USES |
CN112553210A (zh) | 2017-10-03 | 2021-03-26 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
CN108096243B (zh) * | 2017-11-24 | 2020-05-29 | 江苏康缘药业股份有限公司 | 银杏内酯组合物的医药用途 |
WO2019132624A1 (ko) * | 2017-12-29 | 2019-07-04 | 주식회사 헬릭스미스 | 하이브리드 hgf 유전자가 도입된 aav(아데노-연관 바이러스) 벡터 |
AU2019211446A1 (en) * | 2018-01-25 | 2020-09-10 | Biogen Ma Inc. | Methods of treating spinal muscular atrophy |
US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
KR20200108315A (ko) | 2018-02-09 | 2020-09-17 | 제넨테크, 인크. | Tmem106b의 발현을 조절하기 위한 올리고뉴클레오티드 |
BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
CN112334157A (zh) * | 2018-04-17 | 2021-02-05 | 应用干细胞有限公司 | 治疗脊髓肌萎缩症的组合物和方法 |
EP3801638A1 (en) * | 2018-06-08 | 2021-04-14 | Novartis AG | Cell-based assay for measuring drug product potency |
CN108795946B (zh) * | 2018-06-28 | 2022-03-04 | 北京锦篮基因科技有限公司 | 携带设计smn1基因表达框的重组腺相关病毒及应用 |
CN109266682B (zh) * | 2018-09-29 | 2022-04-01 | 中国科学院武汉物理与数学研究所 | 一种神经细胞快速逆行跨突触标记的方法及应用 |
RU2731514C2 (ru) * | 2018-12-21 | 2020-09-03 | Селл энд Джин Терапи Лтд | Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов SHH, CTNNB1, NOG, WNT7A для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-SHH, или Escherichia coli SCS110-AF/VTvaf17-CTNNB1, или Escherichia coli SCS110-AF/VTvaf17-NOG, или Escherichia coli SCS110-AF/VTvaf17-WNT7A, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора |
WO2020160458A1 (en) * | 2019-02-01 | 2020-08-06 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
CA3133453A1 (en) | 2019-03-21 | 2020-09-24 | Daniel Mccoy | Recombinant adeno-associated virus vectors |
CN114174324A (zh) | 2019-04-10 | 2022-03-11 | 普利维尔治疗公司 | 用于溶酶体病症的基因疗法 |
EP3952924A4 (en) * | 2019-04-12 | 2023-05-24 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS OF ADMINISTRATION OF THERAPEUTIC PRODUCTS |
BR112022006530A2 (pt) | 2019-10-17 | 2022-07-05 | Stridebio Inc | Vetores virais adenoassociados para o tratamento da doença de niemann-pick tipo c |
WO2021081201A1 (en) * | 2019-10-22 | 2021-04-29 | Applied Genetic Technologies Corporation | Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegeneative diseases or disorders |
WO2021119622A1 (en) * | 2019-12-12 | 2021-06-17 | Northwestern University | Modulation of ubiquitin carboxy-terminal hydrolase ligase 1 expression |
TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
CN115867650A (zh) | 2020-04-20 | 2023-03-28 | 克里斯蒂安娜基因编辑研究公司 | 用于肺癌治疗的aav递送*** |
CN112121179A (zh) * | 2020-09-15 | 2020-12-25 | 山东兴瑞生物科技有限公司 | 一种组合物及其在治疗脊髓性肌萎缩症中的应用 |
CR20230363A (es) * | 2021-01-29 | 2024-02-20 | Biocad Joint Stock Co | Efecto sinérgico de smn1 y mir-23a en el tratamiento de la atrofia muscular espinal |
WO2023150544A1 (en) * | 2022-02-01 | 2023-08-10 | Seattle Children's Hospital D/B/A Seattle Children's Research Institute | Simplified method of preparing cells for patient administration |
WO2023196841A2 (en) * | 2022-04-05 | 2023-10-12 | The Johns Hopkins University | Methods and materials for treating syngap1-associated neurodevelopmental disorders |
WO2023201207A1 (en) | 2022-04-11 | 2023-10-19 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
KR20230159287A (ko) * | 2022-05-10 | 2023-11-21 | 서울대학교산학협력단 | 인간 smn1 단백질 변이체 및 이의 용도 |
CN114903010B (zh) * | 2022-05-19 | 2024-06-07 | 暨南大学 | 神经退行性疾病模型的构建方法 |
WO2023240236A1 (en) | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
CN118045206A (zh) * | 2024-04-12 | 2024-05-17 | 四川至善唯新生物科技有限公司 | 一种治疗脊髓型肌肉萎缩的药物组合物及其用途 |
Family Cites Families (88)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4703008A (en) | 1983-12-13 | 1987-10-27 | Kiren-Amgen, Inc. | DNA sequences encoding erythropoietin |
NZ210501A (en) | 1983-12-13 | 1991-08-27 | Kirin Amgen Inc | Erythropoietin produced by procaryotic or eucaryotic expression of an exogenous dna sequence |
US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
JPS62171696A (ja) | 1986-01-23 | 1987-07-28 | Sumitomo Chem Co Ltd | ヒトエリスロポエチンの製造方法 |
US4954437A (en) | 1986-09-15 | 1990-09-04 | Integrated Genetics, Inc. | Cell encoding recombinant human erythropoietin |
US5328470A (en) | 1989-03-31 | 1994-07-12 | The Regents Of The University Of Michigan | Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor |
US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
US5605690A (en) | 1989-09-05 | 1997-02-25 | Immunex Corporation | Methods of lowering active TNF-α levels in mammals using tumor necrosis factor receptor |
US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
WO1992001070A1 (en) | 1990-07-09 | 1992-01-23 | The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce | High efficiency packaging of mutant adeno-associated virus using amber suppressions |
US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
US5747469A (en) * | 1991-03-06 | 1998-05-05 | Board Of Regents, The University Of Texas System | Methods and compositions comprising DNA damaging agents and p53 |
DE69233013T2 (de) | 1991-08-20 | 2004-03-04 | The Government Of The United States Of America As Represented By The Secretary Of National Institute Of Health, Office Of Technology Transfer | Adenovirus vermittelter gentransfer in den gastrointestinaltrakt |
US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
US6686200B1 (en) | 1993-08-31 | 2004-02-03 | Uab Research Foundation | Methods and compositions for the large scale production of recombinant adeno-associated virus |
CA2176117C (en) | 1993-11-09 | 2006-01-03 | Terence R. Flotte | Generation of high titers of recombinant aav vectors |
US5837484A (en) | 1993-11-09 | 1998-11-17 | Medical College Of Ohio | Stable cell lines capable of expressing the adeno-associated virus replication gene |
EP0755454B1 (en) | 1994-04-13 | 2008-02-13 | The Rockefeller University | Aav-mediated delivery of dna to cells of the nervous system |
US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
EP0708178A1 (en) | 1994-10-19 | 1996-04-24 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Survival motor neuron (SMN) gene: a gene for spinal muscular atrophy |
WO1996017947A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
US6281010B1 (en) | 1995-06-05 | 2001-08-28 | The Trustees Of The University Of Pennsylvania | Adenovirus gene therapy vehicle and cell line |
US5756283A (en) | 1995-06-05 | 1998-05-26 | The Trustees Of The University Of Pennsylvania | Method for improved production of recombinant adeno-associated viruses for gene therapy |
US5882914A (en) | 1995-06-06 | 1999-03-16 | The Johns Hopkins University School Of Medicine | Nucleic acids encoding the hypoxia inducible factor-1 |
US5677158A (en) | 1995-06-07 | 1997-10-14 | Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
US5622856A (en) | 1995-08-03 | 1997-04-22 | Avigen | High efficiency helper system for AAV vector production |
US6004797A (en) | 1995-11-09 | 1999-12-21 | Avigen, Inc. | Adenovirus helper-free recombinant AAV Virion production |
AU2064297A (en) | 1996-02-29 | 1997-09-16 | Immusol, Inc | Hepatitis c virus ribozymes |
AU1980697A (en) | 1996-03-04 | 1997-09-22 | Targeted Genetics Corporation | Methods for transducing cells in blood vessels using recombinant AAV vec tors |
US20040076613A1 (en) * | 2000-11-03 | 2004-04-22 | Nicholas Mazarakis | Vector system |
US6261823B1 (en) | 1996-12-13 | 2001-07-17 | Schering Corporation | Methods for purifying viruses |
EP0950091A2 (en) | 1996-12-18 | 1999-10-20 | Targeted Genetics Corporation | Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors |
WO1998027207A1 (en) | 1996-12-18 | 1998-06-25 | Targeted Genetics Corporation | Recombinase-activatable aav packaging cassettes for use in the production of aav vectors |
JP2002514914A (ja) | 1997-01-23 | 2002-05-21 | イミュソール インコーポレイテッド | ランダム化または標的特異的リボザイム遺伝子ベクターライブラリーを用いた遺伝子機能の分析および発見 |
US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
AU756827B2 (en) | 1997-10-21 | 2003-01-23 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRs) for use with recombinant AAV vectors |
JP2001520051A (ja) | 1997-10-21 | 2001-10-30 | ターゲテッド ジェネティックス コーポレイション | 組換えaavベクターの産生のための増幅可能アデノ随伴ウイルス(aav)パッケージングカセット |
IL136454A0 (en) | 1997-12-04 | 2001-06-14 | Genzyme Corp | Compositions and methods for inducing gene expression |
KR100912362B1 (ko) | 1998-02-17 | 2009-08-19 | 쉐링 코포레이션 | 바이러스 제제의 정제방법 |
AUPP249298A0 (en) | 1998-03-20 | 1998-04-23 | Ag-Gene Australia Limited | Synthetic genes and genetic constructs comprising same I |
JP2002506652A (ja) | 1998-03-20 | 2002-03-05 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスのヘルパーを含まない製造のための組成物及び方法 |
ATE431418T1 (de) | 1998-05-27 | 2009-05-15 | Genzyme Corp | Aav vektoren zur herstellung der medikamente zur konvektion-erhöhten verabreichung |
EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
DE69939903D1 (de) | 1998-09-04 | 2008-12-24 | Targeted Genetics Corp | Methoden zur herstellung von helpervirusfreien präparationen von freien rekombinenten aav-vektoren mit hohem titer |
US6646113B1 (en) * | 1998-09-17 | 2003-11-11 | The Trustees Of The University Of Pennsylvania | Nucleic acid molecule encoding human survival of motor neuron-interacting protein 1 (SIP1) deletion mutants |
US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
US6893865B1 (en) | 1999-04-28 | 2005-05-17 | Targeted Genetics Corporation | Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles |
CA2374597A1 (en) | 1999-05-28 | 2000-12-07 | Targeted Genetics Corporation | Methods and compositions for lowering the level of tumor necrosis factor (tnf) in the tnf-associated disorders |
JP4827353B2 (ja) | 1999-08-09 | 2011-11-30 | ターゲティッド ジェネティクス コーポレイション | 鎖内塩基対を形成するような配列の設計による、組換えウイルスベクターからの一本鎖の異種ヌクレオチド配列の発現の増大 |
JP5118798B2 (ja) | 2000-03-07 | 2013-01-16 | メルク・シャープ・エンド・ドーム・コーポレイション | アデノウイルス製剤 |
US7125705B2 (en) | 2000-04-28 | 2006-10-24 | Genzyme Corporation | Polynucleotides for use in recombinant adeno-associated virus virion production |
ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
US6593123B1 (en) | 2000-08-07 | 2003-07-15 | Avigen, Inc. | Large-scale recombinant adeno-associated virus (rAAV) production and purification |
US6667174B2 (en) | 2000-09-18 | 2003-12-23 | Genzyme Corporation | Expression vectors containing hybrid ubiquitin promoters |
EP1402043A1 (en) | 2001-07-03 | 2004-03-31 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods of administering vectors to synaptically connected neurons |
US20030050273A1 (en) * | 2001-08-29 | 2003-03-13 | Keiya Ozawa | Compositions and methods for treating neurodegenerative diseases |
AU2002348151A1 (en) | 2001-11-05 | 2003-05-19 | Genvec, Inc. | Viral vector production methods and compositions |
US20030134404A1 (en) | 2001-11-26 | 2003-07-17 | Lochrie Michael A. | Methods for producing stocks of recombinant AAV virions |
AU2002359284A1 (en) | 2001-12-17 | 2003-06-30 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor |
PL1620133T3 (pl) | 2003-05-01 | 2016-05-31 | Genzyme Corp | Terapia genowa dla zaburzeń neurometabolicznych |
US7765583B2 (en) | 2005-02-28 | 2010-07-27 | France Telecom | System and method for managing virtual user domains |
CN107007842A (zh) | 2005-05-02 | 2017-08-04 | 建新公司 | 神经代谢疾病的基因治疗 |
DK1879623T3 (da) * | 2005-05-02 | 2012-12-17 | Genzyme Corp | Genterapi til rygmarvssygdomme |
AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
EP1979485A2 (en) | 2006-01-31 | 2008-10-15 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
US8129510B2 (en) | 2006-03-30 | 2012-03-06 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
CA2654292C (en) | 2006-06-07 | 2022-01-11 | Genzyme Corporation | Gene therapy for motor neuron disorders |
HUE031182T2 (en) | 2006-10-03 | 2017-06-28 | Genzyme Corp | Gene therapy for spinal muscle atrophy |
ITMI20070127A1 (it) | 2007-01-29 | 2008-07-30 | Fond I R C C S | Proteine e-o peptidi per la prevenzione e-o cura di malattie neurodegenerative |
US20080187512A1 (en) * | 2007-02-07 | 2008-08-07 | Academia Sinica | Treatment for spinal muscular atrophy |
EP3121190A1 (en) | 2007-07-14 | 2017-01-25 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
US8633019B2 (en) | 2008-05-27 | 2014-01-21 | Ptc Therapeutics, Inc. | Methods for treating spinal muscular atrophy |
WO2010071832A1 (en) | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
HUE057606T2 (hu) | 2009-05-02 | 2022-05-28 | Genzyme Corp | Génterápia neurodegeneratív rendellenességekre |
ES2684222T3 (es) | 2012-08-01 | 2018-10-01 | Nationwide Children's Hospital | Administración intratecal de virus adenoasociado 9 recombinante |
FR3002237B1 (fr) | 2013-02-15 | 2017-12-15 | Genethon | Methodes pour la production de particules virales aav double brin |
US10821154B2 (en) | 2013-05-01 | 2020-11-03 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
IL292951B1 (en) | 2014-05-02 | 2024-06-01 | Genzyme Corp | AAV vectors for gene therapy in the central nervous system and retina |
-
2010
- 2010-04-27 HU HUE18175397A patent/HUE057606T2/hu unknown
- 2010-04-27 RS RS20220002A patent/RS62779B1/sr unknown
- 2010-04-27 SG SG10201404528SA patent/SG10201404528SA/en unknown
- 2010-04-27 CA CA2759801A patent/CA2759801C/en active Active
- 2010-04-27 SI SI201031748T patent/SI2424991T1/sl unknown
- 2010-04-27 HU HUE10772363A patent/HUE039345T2/hu unknown
- 2010-04-27 RU RU2011149094/10A patent/RU2603740C2/ru not_active IP Right Cessation
- 2010-04-27 RU RU2016140850A patent/RU2743398C2/ru active
- 2010-04-27 SG SG10202109219S patent/SG10202109219SA/en unknown
- 2010-04-27 PT PT181753971T patent/PT3421603T/pt unknown
- 2010-04-27 KR KR1020117028766A patent/KR101835490B1/ko active IP Right Grant
- 2010-04-27 MX MX2016010101A patent/MX356669B/es unknown
- 2010-04-27 MX MX2011011594A patent/MX341073B/es active IP Right Grant
- 2010-04-27 PT PT10772363T patent/PT2424991T/pt unknown
- 2010-04-27 CN CN201080027833.7A patent/CN102459611B/zh active Active
- 2010-04-27 EP EP10772363.7A patent/EP2424991B1/en active Active
- 2010-04-27 HR HRP20212024TT patent/HRP20212024T1/hr unknown
- 2010-04-27 WO PCT/US2010/001239 patent/WO2010129021A1/en active Application Filing
- 2010-04-27 LT LTEP18175397.1T patent/LT3421603T/lt unknown
- 2010-04-27 DK DK10772363.7T patent/DK2424991T3/en active
- 2010-04-27 BR BRPI1010868A patent/BRPI1010868A2/pt not_active Application Discontinuation
- 2010-04-27 EP EP21200213.3A patent/EP3988660A1/en active Pending
- 2010-04-27 EP EP24154262.0A patent/EP4342992A3/en active Pending
- 2010-04-27 SI SI201032101T patent/SI3421603T1/sl unknown
- 2010-04-27 EP EP18175397.1A patent/EP3421603B1/en active Active
- 2010-04-27 SG SG2011079837A patent/SG175409A1/en unknown
- 2010-04-27 LT LTEP10772363.7T patent/LT2424991T/lt unknown
- 2010-04-27 ES ES10772363.7T patent/ES2686504T3/es active Active
- 2010-04-27 JP JP2012508474A patent/JP5879256B2/ja active Active
- 2010-04-27 DK DK18175397.1T patent/DK3421603T3/da active
- 2010-04-27 PL PL10772363T patent/PL2424991T3/pl unknown
- 2010-04-27 KR KR1020187005929A patent/KR20180027619A/ko not_active Application Discontinuation
- 2010-04-27 CN CN201610906309.6A patent/CN107083400A/zh active Pending
- 2010-04-27 ES ES18175397T patent/ES2903127T3/es active Active
- 2010-04-27 PL PL18175397T patent/PL3421603T3/pl unknown
-
2011
- 2011-11-01 MX MX2023006169A patent/MX2023006169A/es unknown
- 2011-11-01 IL IL216094A patent/IL216094A0/en not_active IP Right Cessation
- 2011-11-01 MX MX2018006955A patent/MX2018006955A/es unknown
- 2011-11-02 US US13/287,583 patent/US20130287736A1/en not_active Abandoned
-
2015
- 2015-10-01 JP JP2015195602A patent/JP6397391B2/ja active Active
- 2015-10-06 US US14/876,683 patent/US9415119B2/en active Active
-
2016
- 2016-05-20 US US15/160,949 patent/US10369193B2/en active Active
-
2018
- 2018-06-07 IL IL259898A patent/IL259898A/en unknown
- 2018-06-07 JP JP2018109162A patent/JP2018148927A/ja active Pending
- 2018-09-06 HR HRP20181423TT patent/HRP20181423T1/hr unknown
- 2018-09-06 CY CY181100934T patent/CY1120982T1/el unknown
-
2020
- 2020-02-18 US US16/794,031 patent/US20200384076A1/en not_active Abandoned
-
2022
- 2022-10-13 US US18/046,363 patent/US11975043B2/en active Active
- 2022-11-10 US US18/054,500 patent/US11911440B2/en active Active
-
2023
- 2023-10-17 US US18/488,346 patent/US20240033323A1/en active Pending
Also Published As
Similar Documents
Publication | Publication Date | Title |
---|---|---|
MX2023006169A (es) | Terapia genica para trastornos neurodegenerativos. | |
WO2010048273A3 (en) | Methods and compounds for treatment of neurodegenerative disorders | |
WO2012036433A3 (ko) | miRNA를 타겟으로 한 신경퇴행성 질환 치료 | |
TN2010000140A1 (en) | Compositions and methods for use antibodies against sclerostin | |
HRP20130345T1 (hr) | NOVI TERAPIJSKI PRISTUPI ZA LIJEČENJE CMT-a I SRODNIH POREMEĆA | |
WO2010071826A3 (en) | Methods for treating osteoclast-related disease, compounds and compositions thereof | |
WO2011069063A3 (en) | Multicyclic compounds and methods of use thereof | |
WO2011082273A3 (en) | Substituted pyrrolo-aminopyrimidine compounds | |
WO2012075492A3 (en) | Carbocycle-substituted purine and 7-deazapurine compounds | |
GB2463833A (en) | Methods and compositions for the treatment of neurological disorders | |
WO2014093114A8 (en) | Ppar-sparing thiazolidinediones and combinations for the treatment of neurodegenerative diseases | |
WO2011082267A3 (en) | Substituted triazolo-pyrazine compounds | |
EA201290498A1 (ru) | Терапевтические полимерные наночастицы, включающие эпотилон, и способы их получения и применения | |
WO2011005595A3 (en) | 2-5a analogs and their methods of use | |
MY161818A (en) | Styrenyl derivate compounds for treating ophthalmic diseases and disorders | |
MX2012004525A (es) | Compuestos. | |
WO2011083150A3 (en) | Obesity small molecules | |
WO2012149478A3 (en) | Agents useful for treating friedreich's ataxia and other neurodegenerative diseases | |
WO2010075280A3 (en) | Coumarin-based compounds for the treatment of alzheimer's disease and cancer | |
PL2384339T3 (pl) | Leczenie choroby autoimmunologicznej przez modulowanie aneksyny-1 (lipokortyny 1) | |
EA201400969A1 (ru) | Новые терапевтические подходы для лечения болезни паркинсона | |
WO2011053901A3 (en) | Magnetic nanostructures as theranostic agents | |
WO2010067078A8 (en) | 3,6-disubstituted xanthylium salts | |
EP2139460A4 (en) | COMPOSITIONS AND METHODS FOR THE DIAGNOSIS, TREATMENT AND PREVENTION OF AMYOTROPHER LATERAL SCLEROSIS AND ASSOCIATED NEUROLOGICAL DISEASES | |
MX2010010303A (es) | Nuevos compuestos de acido ribonucleico de pequeña interferencia para inhibir rtp801. |