PT1778297E - Vector viral adenoassociado para efectuar salto de exões num gene que codifica para uma proteína com domínios dispensáveis - Google Patents
Vector viral adenoassociado para efectuar salto de exões num gene que codifica para uma proteína com domínios dispensáveis Download PDFInfo
- Publication number
- PT1778297E PT1778297E PT05797742T PT05797742T PT1778297E PT 1778297 E PT1778297 E PT 1778297E PT 05797742 T PT05797742 T PT 05797742T PT 05797742 T PT05797742 T PT 05797742T PT 1778297 E PT1778297 E PT 1778297E
- Authority
- PT
- Portugal
- Prior art keywords
- adeno
- dispensable
- sequence
- associated viral
- gene encoding
- Prior art date
Links
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
- 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
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Veterinary Medicine (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
FR0451861A FR2874384B1 (fr) | 2004-08-17 | 2004-08-17 | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
Publications (1)
Publication Number | Publication Date |
---|---|
PT1778297E true PT1778297E (pt) | 2009-07-23 |
Family
ID=34948645
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PT05797742T PT1778297E (pt) | 2004-08-17 | 2005-08-02 | Vector viral adenoassociado para efectuar salto de exões num gene que codifica para uma proteína com domínios dispensáveis |
Country Status (12)
Country | Link |
---|---|
US (2) | US20120077860A1 (pt) |
EP (2) | EP1778297B1 (pt) |
JP (1) | JP4885136B2 (pt) |
AT (1) | ATE429252T1 (pt) |
CA (1) | CA2576462C (pt) |
DE (1) | DE602005014114D1 (pt) |
DK (1) | DK1778297T3 (pt) |
ES (1) | ES2324948T3 (pt) |
FR (1) | FR2874384B1 (pt) |
PL (1) | PL1778297T3 (pt) |
PT (1) | PT1778297E (pt) |
WO (1) | WO2006021724A2 (pt) |
Families Citing this family (64)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
SI1766010T1 (sl) | 2004-06-28 | 2011-06-30 | Univ Western Australia | Protismiselni oligonukleotidi za induciranje preskakovanja eksonov in postopki za njihovo uporabo |
WO2008113773A2 (en) * | 2007-03-16 | 2008-09-25 | Biorigen S.R.L. | Gene expression regulation technology and noncoding rnas for diagnosis and therapy |
CA3066050A1 (en) | 2008-10-24 | 2010-04-29 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
JP2012523225A (ja) * | 2009-04-10 | 2012-10-04 | アソシアシオン・アンスティテュ・ドゥ・ミオロジー | 疾患の処置のためのトリシクロ−dnaアンチセンスオリゴヌクレオチド、組成物及び方法 |
PL2499249T3 (pl) | 2009-11-12 | 2019-03-29 | Univ Western Australia | Cząsteczki antysensowne i sposoby leczenia patologii |
EP2524037B1 (en) | 2010-01-12 | 2018-05-16 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
ES2566553T3 (es) | 2010-03-17 | 2016-04-13 | Association Institut De Myologie | ARNnp U7 modificados para el tratamiento de las enfermedades neuromusculares |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
WO2012109570A1 (en) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
EP2500434A1 (en) | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
CA2834729A1 (en) | 2011-04-06 | 2012-10-11 | Universite Pierre Et Marie Curie (Paris 6) | A skeletal muscle-specific enhancer |
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EA202090946A3 (ru) | 2013-03-14 | 2021-07-30 | Сарепта Терапьютикс, Инк. | Композиции, обеспечивающие пропускание экзонов, для лечения мышечной дистрофии |
EP3662912A1 (en) | 2013-03-15 | 2020-06-10 | Sarepta Therapeutics, Inc. | Improved dosages of eteplirsen for treating duchenne muscular dystrophy |
AU2014227766B2 (en) | 2013-03-15 | 2018-10-04 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding AAV vectors |
EP3517612A1 (en) | 2013-03-15 | 2019-07-31 | The University of North Carolina At Chapel Hill | Synthetic adeno-associated virus inverted terminal repeats |
JP6461917B2 (ja) | 2013-04-20 | 2019-01-30 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | エクソン2標的U7snRNAポリヌクレオチド構築物の組換えアデノ随伴ウイルスによる送達 |
CN105339012A (zh) | 2013-05-03 | 2016-02-17 | 西莱克塔生物科技公司 | 降低i型和iv型超敏反应的致耐受性合成纳米载体的局部伴随施用 |
US10059947B2 (en) | 2013-09-11 | 2018-08-28 | Synthena Ag | Nucleic acids and methods for the treatment of Pompe disease |
HRP20220379T1 (hr) * | 2014-06-10 | 2022-05-27 | Erasmus University Medical Center Rotterdam | Antisense oligonukleotidi korisni u liječenju pompeove bolesti |
EP3174981B1 (en) | 2014-07-31 | 2020-03-25 | Association Institut de Myologie | Treatment of amyotrophic lateral sclerosis |
AU2015301978C1 (en) | 2014-08-09 | 2022-06-09 | Research Institute At Nationwide Children's Hospital | Methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene |
CA2957800A1 (en) | 2014-09-07 | 2016-03-10 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector immune responses |
RU2727015C2 (ru) | 2014-11-21 | 2020-07-17 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Векторы aav, нацеленные на центральную нервную систему |
CA2972807C (en) | 2015-01-14 | 2024-01-16 | The University Of North Carolina At Chapel Hill | Methods and compositions for targeted gene transfer |
WO2016134300A1 (en) | 2015-02-20 | 2016-08-25 | Board Of Regents, The University Of Texas System | Methods and compositions for attenuated chlamydia as vaccine and vector |
CA2999192A1 (en) | 2015-09-21 | 2017-03-30 | Association Institut De Myologie | Antisense oligonucleotides hybridizing with a key element of the polyadenylation region of a dux4 pre-mrna and uses thereof |
CA2996420A1 (en) | 2015-09-28 | 2017-04-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
FR3044926B1 (fr) * | 2015-12-09 | 2020-01-31 | Genethon | Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine |
WO2018169811A1 (en) | 2017-03-11 | 2018-09-20 | Selecta Biosciences, Inc. | Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant |
JP7132934B2 (ja) | 2017-03-15 | 2022-09-07 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | ポリプロイド性アデノ随伴ウイルスベクターならびにその作製および使用の方法 |
GB201711809D0 (en) * | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
KR20210006358A (ko) | 2018-04-03 | 2021-01-18 | 스트라이드바이오 인코포레이티드 | 안 조직을 표적으로 하기 위한 바이러스 벡터 |
CA3095179A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
JP7406677B2 (ja) | 2018-04-03 | 2023-12-28 | ギンコ バイオワークス インコーポレイテッド | 抗体を回避するウイルスベクター |
EP4219717A3 (en) * | 2018-06-13 | 2023-12-20 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
CN112639110A (zh) | 2018-06-22 | 2021-04-09 | 阿斯克肋匹奥生物制药公司 | 用于基因递送以在细胞内持续存在的载体 |
KR20210081324A (ko) | 2018-08-02 | 2021-07-01 | 다인 세라퓨틱스, 인크. | 근육 표적화 복합체 및 안면견갑상완 근육 이영양증을 치료하기 위한 그의 용도 |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
TW202102525A (zh) | 2019-03-21 | 2021-01-16 | 美商史崔德生物公司 | 重組腺相關病毒載體 |
CN113767110A (zh) | 2019-04-26 | 2021-12-07 | 北卡罗来纳-查佩尔山大学 | 用于双重聚糖结合aav2.5载体的方法和组合物 |
KR102452590B1 (ko) * | 2019-08-01 | 2022-10-12 | 기초과학연구원 | CRISPR/Cas9 매개 체세포 핵 이식에 의한 개 디스트로핀병증 모델 생성 및 이의 용도 |
TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療c型尼曼—匹克病之腺相關病毒載體 |
JP2023500884A (ja) | 2019-11-06 | 2023-01-11 | アソシエーション・アンスティトゥート・ドゥ・マイオロジー | 筋疾患のための併用療法 |
WO2021205005A2 (en) | 2020-04-09 | 2021-10-14 | Association Institut De Myologie | Antisense sequences for treating amyotrophic lateral sclerosis |
EP4188404A1 (en) * | 2020-07-29 | 2023-06-07 | The Board Of Regents Of The University Of Texas System | Transgene cassettes, aav vectors, and aav viral vectors for expression of human codon-optimized cstb |
US20230357795A1 (en) | 2020-09-15 | 2023-11-09 | Research Institute At Nationwide Children's Hospital | Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy |
WO2022093769A1 (en) | 2020-10-28 | 2022-05-05 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding aav2.5 vector |
AU2021369480A1 (en) | 2020-10-28 | 2023-04-20 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding AAV2.5 vector |
TW202242124A (zh) | 2021-01-14 | 2022-11-01 | 美商史崔德生物公司 | 靶向t細胞之aav載體 |
WO2022155578A1 (en) | 2021-01-15 | 2022-07-21 | Outpace Bio, Inc. | Small molecule-regulated gene expression system |
JP2024508324A (ja) | 2021-03-04 | 2024-02-26 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | Dmdエクソンの重複を修正するためのcrispr-cas9を使用したジストロフィンベースのミオパチーの治療のための生成物及び方法 |
US20240189447A1 (en) | 2021-04-16 | 2024-06-13 | Asklepios Biopharmaceutical, Inc. | Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response |
JP2024515720A (ja) | 2021-04-23 | 2024-04-10 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィーを治療するための製品及び方法 |
US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
US11771776B2 (en) * | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
EP4119682A1 (en) | 2021-07-13 | 2023-01-18 | Genethon | A method for determining the nature of dna impurities inside viral particles |
CA3230419A1 (en) * | 2021-09-03 | 2023-03-09 | David A. Nelles | Rna editing via recruitment of spliceosome components |
EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
WO2023150687A1 (en) | 2022-02-04 | 2023-08-10 | Ginkgo Bioworks, Inc. | Recombinant adeno-associated virus vectors, and methods of use thereof |
WO2024124019A2 (en) | 2022-12-07 | 2024-06-13 | Ginkgo Bioworks, Inc. | Aav vectors targeting hematopoietic stem cells |
Family Cites Families (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP2000325085A (ja) | 1999-05-21 | 2000-11-28 | Masafumi Matsuo | デュシェンヌ型筋ジストロフィー治療剤 |
ES2308989T3 (es) * | 1999-08-09 | 2008-12-16 | Targeted Genetics Corporation | Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios. |
US7638120B2 (en) * | 2000-03-14 | 2009-12-29 | Thomas Jefferson University | High transgene expression of a pseudotyped adeno-associated virus type |
US6653467B1 (en) * | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
US6727355B2 (en) | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
EP1191097A1 (en) * | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
CN1620508A (zh) * | 2001-12-21 | 2005-05-25 | 牛津生物医学(英国)有限公司 | 转基因生物 |
ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
AU2003225410A1 (en) * | 2003-03-21 | 2004-10-11 | Academisch Ziekenhuis Leiden | Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure |
-
2004
- 2004-08-17 FR FR0451861A patent/FR2874384B1/fr active Active
-
2005
- 2005-08-02 AT AT05797742T patent/ATE429252T1/de active
- 2005-08-02 WO PCT/FR2005/050642 patent/WO2006021724A2/fr active Application Filing
- 2005-08-02 CA CA2576462A patent/CA2576462C/fr active Active
- 2005-08-02 US US11/573,811 patent/US20120077860A1/en not_active Abandoned
- 2005-08-02 ES ES05797742T patent/ES2324948T3/es active Active
- 2005-08-02 EP EP05797742A patent/EP1778297B1/fr active Active
- 2005-08-02 EP EP08163844A patent/EP2014309A1/fr not_active Withdrawn
- 2005-08-02 PT PT05797742T patent/PT1778297E/pt unknown
- 2005-08-02 DK DK05797742T patent/DK1778297T3/da active
- 2005-08-02 JP JP2007526520A patent/JP4885136B2/ja active Active
- 2005-08-02 DE DE602005014114T patent/DE602005014114D1/de active Active
- 2005-08-02 PL PL05797742T patent/PL1778297T3/pl unknown
-
2012
- 2012-04-27 US US13/458,037 patent/US20130072541A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
DE602005014114D1 (de) | 2009-06-04 |
DK1778297T3 (da) | 2009-07-27 |
FR2874384B1 (fr) | 2010-07-30 |
US20130072541A1 (en) | 2013-03-21 |
EP1778297A2 (fr) | 2007-05-02 |
CA2576462A1 (fr) | 2006-03-02 |
JP2008509695A (ja) | 2008-04-03 |
US20120077860A1 (en) | 2012-03-29 |
PL1778297T3 (pl) | 2009-10-30 |
EP1778297B1 (fr) | 2009-04-22 |
EP2014309A1 (fr) | 2009-01-14 |
ATE429252T1 (de) | 2009-05-15 |
FR2874384A1 (fr) | 2006-02-24 |
JP4885136B2 (ja) | 2012-02-29 |
WO2006021724A2 (fr) | 2006-03-02 |
WO2006021724A3 (fr) | 2006-08-31 |
CA2576462C (fr) | 2013-03-12 |
ES2324948T3 (es) | 2009-08-20 |
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