WO2015153760A3 - Methods and compositions for prevention or treatment of a nervous system disorder - Google Patents

Methods and compositions for prevention or treatment of a nervous system disorder Download PDF

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Publication number
WO2015153760A3
WO2015153760A3 PCT/US2015/023882 US2015023882W WO2015153760A3 WO 2015153760 A3 WO2015153760 A3 WO 2015153760A3 US 2015023882 W US2015023882 W US 2015023882W WO 2015153760 A3 WO2015153760 A3 WO 2015153760A3
Authority
WO
WIPO (PCT)
Prior art keywords
nervous system
compositions
methods
protein
prevention
Prior art date
Application number
PCT/US2015/023882
Other languages
French (fr)
Other versions
WO2015153760A2 (en
Inventor
Fyodor Urnov
H. Steve Zhang
Original Assignee
Sangamo Biosciences, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sangamo Biosciences, Inc. filed Critical Sangamo Biosciences, Inc.
Publication of WO2015153760A2 publication Critical patent/WO2015153760A2/en
Publication of WO2015153760A3 publication Critical patent/WO2015153760A3/en

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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Methods and compositions for nervous system (NS) disorders are provided. Specifically, the disclosure provides methods and compositions for altering the expression of, or for correcting, one or more genes whose gene products are involved in a nervous system (NS) disorder such as a disease or disorder of the central nervous system (CNS) and/or peripheral nervous system (PNS) by administering a transcription factor or nuclease that targets the endogenous'gene, wherein the transcription factor or nuclease is selected from the group consisting of a protein comprising a zinc finger protein, a protein comprising a TAL-effector protein and a CRISPR/Cas system.
PCT/US2015/023882 2014-04-01 2015-04-01 Methods and compositions for prevention or treatment of a nervous system disorder WO2015153760A2 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201461973471P 2014-04-01 2014-04-01
US61/973,471 2014-04-01
US201461992656P 2014-05-13 2014-05-13
US61/992,656 2014-05-13

Publications (2)

Publication Number Publication Date
WO2015153760A2 WO2015153760A2 (en) 2015-10-08
WO2015153760A3 true WO2015153760A3 (en) 2015-11-26

Family

ID=54241433

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2015/023882 WO2015153760A2 (en) 2014-04-01 2015-04-01 Methods and compositions for prevention or treatment of a nervous system disorder

Country Status (1)

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WO (1) WO2015153760A2 (en)

Families Citing this family (36)

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EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
CN106459995B (en) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 CRISPR-associated methods and compositions using dominant grnas
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3302048B1 (en) 2015-05-29 2020-02-19 Regeneron Pharmaceuticals, Inc. Non-human animals having a disruption in a c9orf72 locus
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and uses thereof
CN109312339B (en) * 2015-12-23 2022-01-28 克里斯珀医疗股份公司 Materials and methods for treating amyotrophic lateral sclerosis and/or frontotemporal lobar degeneration
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
EP3474876A4 (en) * 2016-06-22 2020-01-15 City of Hope Treatment of canavan disease
SG11201900907YA (en) 2016-08-03 2019-02-27 Harvard College Adenosine nucleobase editors and uses thereof
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
SG10202102997UA (en) 2016-09-30 2021-04-29 Regeneron Pharma Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus
GB2573062A (en) 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
CN110214184A (en) * 2016-12-01 2019-09-06 桑格摩生物治疗股份有限公司 Tau protein regulator and the method and composition delivered for it
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
JP2020534795A (en) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ Methods and Compositions for Evolving Base Editing Factors Using Phage-Supported Continuous Evolution (PACE)
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
CN111819281A (en) * 2017-10-23 2020-10-23 普利维尔治疗公司 Gene therapy for neurodegenerative diseases
CA3079727A1 (en) * 2017-10-24 2019-05-02 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of rare diseases
WO2019109051A1 (en) * 2017-12-01 2019-06-06 Encoded Therapeutics, Inc. Engineered dna binding proteins
MX2021006253A (en) * 2018-11-28 2021-09-21 Prevail Therapeutics Inc Gene therapies for neurodegenerative disease.
JP7449291B2 (en) 2018-12-20 2024-03-13 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Nuclease-mediated repeat expansion
WO2020191153A2 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2021049497A1 (en) * 2019-09-09 2021-03-18 国立大学法人富山大学 Model animal for bipolar disorder
KR20230019843A (en) 2020-05-08 2023-02-09 더 브로드 인스티튜트, 인코퍼레이티드 Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence

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Publication number Priority date Publication date Assignee Title
US20110023153A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in alzheimer's disease

Non-Patent Citations (2)

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WOODRUFF ET AL.: "The Presenilin-1 DeltaE9 mutation results in reduced gamma-secretase activity, but not total loss of PS1 function, in isogenic human stem cells.", CELL REP., vol. 5, no. 4, 2013, pages 974 - 85, XP055238289 *

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