CA2942515C - Raav-based compositions and methods for treating amyotrophic lateral sclerosis - Google Patents

Raav-based compositions and methods for treating amyotrophic lateral sclerosis

Info

Publication number
CA2942515C
CA2942515C CA2942515A CA2942515A CA2942515C CA 2942515 C CA2942515 C CA 2942515C CA 2942515 A CA2942515 A CA 2942515A CA 2942515 A CA2942515 A CA 2942515A CA 2942515 C CA2942515 C CA 2942515C
Authority
CA
Canada
Prior art keywords
nucleic acid
cell
c9orf72
seq
raav
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CA2942515A
Other languages
English (en)
French (fr)
Other versions
CA2942515A1 (en
Inventor
Christian Mueller
Jr. Brown Robert H.
Original Assignee
University of Massachusetts Amherst
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 University of Massachusetts Amherst filed Critical University of Massachusetts Amherst
Publication of CA2942515A1 publication Critical patent/CA2942515A1/en
Application granted granted Critical
Publication of CA2942515C publication Critical patent/CA2942515C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • 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
    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pain & Pain Management (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CA2942515A 2014-03-18 2015-03-18 Raav-based compositions and methods for treating amyotrophic lateral sclerosis Active CA2942515C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461955189P 2014-03-18 2014-03-18
US61/955,189 2014-03-18
PCT/US2015/021321 WO2015143078A1 (en) 2014-03-18 2015-03-18 Raav-based compositions and methods for treating amyotrophic lateral sclerosis

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CA3276269 Division 2015-03-18

Publications (2)

Publication Number Publication Date
CA2942515A1 CA2942515A1 (en) 2015-09-24
CA2942515C true CA2942515C (en) 2025-12-09

Family

ID=54145287

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2942515A Active CA2942515C (en) 2014-03-18 2015-03-18 Raav-based compositions and methods for treating amyotrophic lateral sclerosis

Country Status (8)

Country Link
US (6) US10280418B2 (enExample)
EP (3) EP3750907A3 (enExample)
JP (4) JP6756700B2 (enExample)
AU (3) AU2015231294B2 (enExample)
CA (1) CA2942515C (enExample)
DK (1) DK3119797T3 (enExample)
ES (1) ES2856403T3 (enExample)
WO (1) WO2015143078A1 (enExample)

Families Citing this family (63)

* Cited by examiner, † Cited by third party
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
ES2616051T3 (es) 2008-12-02 2017-06-09 Wave Life Sciences Japan, Inc. Método para la síntesis de ácidos nucleicos modificados en el átomo de fósforo
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
ES2605305T3 (es) 2010-04-23 2017-03-13 University Of Massachusetts Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
EP3318634A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations
JP6128529B2 (ja) 2011-07-19 2017-05-17 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． 官能化核酸の合成のための方法
US9598458B2 (en) 2012-07-13 2017-03-21 Wave Life Sciences Japan, Inc. Asymmetric auxiliary group
DK3094728T3 (da) 2014-01-16 2022-05-16 Wave Life Sciences Ltd Kiralt design
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
EP3750907A3 (en) 2014-03-18 2021-04-28 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
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
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
IL284949B2 (en) 2014-11-14 2024-06-01 Voyager Therapeutics Inc Modulatory polynucleotides
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
BR112017025507A2 (pt) 2015-05-29 2018-08-07 Regeneron Pharmaceuticals, Inc. roedor, célula isolada de roedor ou tecido isolado de roedor, célula-tronco embrionária de roedor, embrião de roedor, e, métodos para preparação de um roedor e para identificação de um candidato terapêutico.
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
WO2017136536A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
EP3432933B1 (en) 2016-03-22 2022-10-19 President and Fellows of Harvard College Biocompatible adhesives and methods of use thereof
CA3018224A1 (en) * 2016-03-31 2017-10-05 University Of Cincinnati Methods and compositions for the treatment of als
IL262056B2 (en) * 2016-04-02 2023-10-01 Res Inst Nationwide Childrens Hospital The U6 precursor system is optimized for tissue-specific expression
EP3440210A4 (en) 2016-04-05 2019-11-27 University of Massachusetts COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
EP3458588B1 (en) 2016-05-18 2025-10-29 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2017214471A1 (en) * 2016-06-10 2017-12-14 Saunders, Ann M. Methods for detecting structural variants in neurodegenerative disease
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
WO2018064600A1 (en) * 2016-09-30 2018-04-05 Regeneron Pharmaceuticals, Inc. Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
CA3043489A1 (en) 2016-11-21 2018-05-24 Nanostring Technologies, Inc. Chemical compositions and methods of using same
CN118910166A (zh) * 2017-05-05 2024-11-08 沃雅戈治疗公司 调节性多核苷酸
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CA3059213A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
US20210108199A1 (en) * 2017-09-06 2021-04-15 The Trustees Of Columbia University In The City Of New York Treatment for aggressive cancers by targeting C9ORF72
AU2018338188B2 (en) 2017-09-22 2025-02-20 University Of Massachusetts SOD1 dual expression vectors and uses thereof
CN111479924B (zh) 2017-10-16 2024-06-14 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
KR20200075865A (ko) * 2017-10-23 2020-06-26 프리베일 테라퓨틱스, 인크. 신경변성 질환에 대한 유전자 요법
WO2019195304A1 (en) * 2018-04-03 2019-10-10 Academia Sinica Mir-17~92 as therapeutic or diagnostic target of motor neuron (mn) degeneration diseases
WO2019222178A1 (en) 2018-05-14 2019-11-21 Nanostring Technologies, Inc. Chemical compositions and methods of using same
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
WO2020053258A1 (en) * 2018-09-12 2020-03-19 Uniqure Ip B.V. Rnai induced c9orf72 suppression for the treatment of als/ftd
MX2021006359A (es) * 2018-11-30 2021-08-11 Novartis Ag Vectores virales de vaa y sus usos.
IL301193A (en) 2018-12-20 2023-05-01 Regeneron Pharma Nuclease-mediated repeat expansion
WO2021067613A1 (en) * 2019-10-01 2021-04-08 Children's Medical Center Corporation Compositions and methods for treating amyotrophic lateral sclerosis
CN114585370A (zh) * 2019-10-06 2022-06-03 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
JP2023512688A (ja) * 2020-02-03 2023-03-28 ラトガース，ザ ステート ユニバーシティ オブ ニュー ジャージー 脊髄損傷後の機能回復を促進するためのマイクロｒｎａ－７組成物およびその使用方法
WO2021159008A2 (en) 2020-02-07 2021-08-12 Maze Therapeutics, Inc. Compositions and methods for treating neurodegenerative diseases
EP4214324A1 (en) * 2020-09-16 2023-07-26 Biogen MA Inc. Compositions and methods for treating amyotrophic lateral sclerosis (als) with aav-mir-sod1
CA3210763A1 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
KR20240004467A (ko) 2021-04-06 2024-01-11 메이즈 테라퓨틱스, 인코퍼레이티드 Tdp-43 단백질병증을 치료하기 위한 조성물 및 방법
US12234472B2 (en) 2021-10-18 2025-02-25 Regeneron Pharmaceuticals, Inc. Eukaryotic cells comprising adenovirus-associated virus polynucleotides
WO2024077109A1 (en) 2022-10-05 2024-04-11 Maze Therapeutics, Inc. Unc13a antisense oligonucleotides and uses thereof

Family Cites Families (159)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
US5252334A (en) 1989-09-08 1993-10-12 Cygnus Therapeutic Systems Solid matrix system for transdermal drug delivery
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5697899A (en) 1995-02-07 1997-12-16 Gensia Feedback controlled drug delivery system
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5656016A (en) 1996-03-18 1997-08-12 Abbott Laboratories Sonophoretic drug delivery system
US5797898A (en) 1996-07-02 1998-08-25 Massachusetts Institute Of Technology Microchip drug delivery devices
US5783208A (en) 1996-07-19 1998-07-21 Theratech, Inc. Transdermal drug delivery matrix for coadministering estradiol and another steroid
US5779708A (en) 1996-08-15 1998-07-14 Cyberdent, Inc. Intraosseous drug delivery device and method
US6177403B1 (en) 1996-10-21 2001-01-23 The Trustees Of The University Of Pennsylvania Compositions, methods, and apparatus for delivery of a macromolecular assembly to an extravascular tissue of an animal
US6544523B1 (en) 1996-11-13 2003-04-08 Chiron Corporation Mutant forms of Fas ligand and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
ATE402254T1 (de) 1998-05-28 2008-08-15 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
US20030110526A1 (en) 1998-08-25 2003-06-12 Robert H. Brown Dysferlin mutations
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・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無しの生産のための組成物および方法
US6544786B1 (en) 1999-10-15 2003-04-08 University Of Pittsburgh Of The Commonwealth Of Higher Education Method and vector for producing and transferring trans-spliced peptides
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
US8202846B2 (en) 2000-03-16 2012-06-19 Cold Spring Harbor Laboratory Methods and compositions for RNA interference
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
EP2221376B1 (en) 2001-06-21 2012-11-21 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US20050019915A1 (en) 2001-06-21 2005-01-27 Bennett C. Frank Antisense modulation of superoxide dismutase 1, soluble expression
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
CA2921821A1 (en) 2001-07-12 2003-01-23 University Of Massachusetts In vivo production of small interfering rnas that mediate gene silencing
EP1419245A4 (en) 2001-07-13 2006-04-05 Univ Iowa Res Found ADENO-ASSOCIATED PSEUDOTYPE VIRUSES AND USES THEREOF
IL161827A0 (en) 2001-11-13 2005-11-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby
EP1463805B1 (en) 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
US20050137153A1 (en) 2002-02-20 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
US20060292117A1 (en) 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
WO2003089011A1 (en) 2002-04-19 2003-10-30 University Of Florida rAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES
ES2280694T3 (es) 2002-04-29 2007-09-16 The Trustees Of The University Of Pennsylvania Metodo para la extraccion directa y la amplificacion de virus integrados del adn celular de tejidos.
US20060093589A1 (en) 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
US20050255086A1 (en) 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US8729036B2 (en) 2002-08-07 2014-05-20 University Of Massachusetts Compositions for RNA interference and methods of use thereof
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
JP2006507841A (ja) 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
AU2004208031B2 (en) 2003-01-28 2009-10-08 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof.
US20040219528A1 (en) 2003-04-15 2004-11-04 Morris David W. Novel therapeutic targets in cancer
US7387896B2 (en) 2003-03-26 2008-06-17 The Regents Of The University Of Michigan MicroRNA vectors
US20060228800A1 (en) 2003-05-15 2006-10-12 Shi-Lung Lin Novel Transgenic Methods Using intronic RNA
US7750144B2 (en) 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
AU2004248136B2 (en) 2003-06-02 2011-09-15 University Of Massachusetts Methods and compositions for controlling efficacy of RNA silencing
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
EP3211085B1 (en) 2003-09-30 2021-03-31 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US20060134787A1 (en) 2004-12-22 2006-06-22 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
WO2005062937A2 (en) 2003-12-22 2005-07-14 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended sirna
CN103060324B (zh) 2004-03-05 2015-04-01 贝尼泰克生物制药有限公司 用于RNAi试剂同时递送的多启动子表达盒
US7498316B2 (en) 2004-04-06 2009-03-03 University Of Massachusetts Methods and compositions for treating gain-of-function disorders using RNA interference
CA2566286A1 (en) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
WO2005116224A2 (en) 2004-05-18 2005-12-08 Children's Memorial Hospital Tetracycline-regulated adeno-associated viral (aav) vectors for gene delivery to the nervous system
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
US20060189564A1 (en) 2004-10-22 2006-08-24 Medtronic, Inc. Methods and sequences to suppress pro-inflamatory cytokine actions locally to treat pain
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
US20060229268A1 (en) * 2004-12-16 2006-10-12 Alsgen, Llc Small interference RNA (siRNA) molecules for modulating superoxide dismutase (SOD)
CN104293835B (zh) 2005-04-07 2017-07-04 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
PL2002003T3 (pl) 2005-05-27 2016-06-30 Ospedale San Raffaele Srl Wektor genetyczny zawierający mi-RNA
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
EP1931782B2 (en) 2005-08-29 2016-04-20 Regulus Therapeutics Inc Methods for use in modulating mir-122a
US9150882B2 (en) 2006-01-31 2015-10-06 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
EP2502999A3 (en) 2006-03-03 2013-01-23 Amorfix Life Sciences Ltd. Methods and compositions to treat and detect misfolded-SOD1 mediated diseases
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
ES2434169T3 (es) 2006-11-08 2013-12-13 Veritas Bio, Llc Entrega in vivo de ARN bicatenario a una célula objetivo
WO2008125846A2 (en) 2007-04-12 2008-10-23 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement
US9353368B2 (en) 2007-05-23 2016-05-31 Ge Healthcare Dharmacon, Inc. Micro-RNA scaffolds and non-naturally occurring micro-RNAs
AU2008260103B2 (en) 2007-05-31 2014-04-03 University Of Iowa Research Foundation Reduction of off-target RNA interference toxicity
WO2009079592A2 (en) 2007-12-17 2009-06-25 California Institute Of Technology Modulating immune system development and function through microrna mir-146
JP5697993B2 (ja) 2008-02-11 2015-04-08 アールエックスアイ ファーマシューティカルズ コーポレーション 修飾ＲＮＡｉポリヌクレオチドおよびその使用
US20090215879A1 (en) 2008-02-26 2009-08-27 University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
CA2717792A1 (en) 2008-03-07 2009-09-11 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
WO2009146178A1 (en) 2008-04-15 2009-12-03 President And Fellows Of Harvard College Angiogenin and amyotrophic lateral sclerosis
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2009149182A1 (en) 2008-06-04 2009-12-10 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small rna targeting of gene promoters
EP2297185A1 (en) 2008-06-17 2011-03-23 Amsterdam Molecular Therapeutics (AMT) B.V. Parvoviral capsid with incorporated gly-ala repeat region
EP2310507A4 (en) 2008-07-08 2013-03-20 David Gladstone Inst METHOD AND COMPOSITIONS FOR ANGIOGENIC MODULATION
WO2010027446A2 (en) 2008-09-04 2010-03-11 Galenea Corp., Et Al. Synaptic vesicle cycling assays and systems
WO2010034314A1 (en) 2008-09-24 2010-04-01 Aarhus Universitet Retroviral delivery of synthectic gene cassettes
WO2010071454A1 (en) 2008-12-17 2010-06-24 Auckland Uniservices Limited Adeno-associated viral vectors and uses thereof
US20110305772A1 (en) 2009-02-26 2011-12-15 The Johns Hopkins University Compositions and methods for ex vivo hepatic nucleic acid delivery
SG175409A1 (en) 2009-05-02 2011-12-29 Genzyme Corp Gene therapy for neurodegenerative disorders
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
CN102695526A (zh) 2009-11-09 2012-09-26 吉恩波多治疗股份公司 用于神经元特异性的体内连续dopa合成的新型病毒载体构建体
US9493776B2 (en) 2009-11-19 2016-11-15 National University Corporation Okayama University System for increasing gene expression and vector comprising the system
WO2011088081A1 (en) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
ES2680915T3 (es) 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
AU2011223820B2 (en) 2010-03-01 2016-01-14 The Children's Hospital Of Philadelphia Nucleic acids for targeting multiple regions of the HCV genome
ES2605305T3 (es) 2010-04-23 2017-03-13 University Of Massachusetts Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
WO2011135396A1 (en) * 2010-04-30 2011-11-03 Cellectis Method for modulating double-strand break-induced homologous recombination
US8785413B2 (en) 2010-09-27 2014-07-22 Florida Atlantic University Research Corporation Materials and methods for the treatment of pathological neovascularization in the eye
US9181544B2 (en) 2011-02-12 2015-11-10 University Of Iowa Research Foundation Therapeutic compounds
WO2012142434A2 (en) 2011-04-15 2012-10-18 THE UNITED STATES OF AMERICA, as represented by the secretary, Depart.of Healtth and Human Services Aav mediated ctla-4 gene transfer to treat sjogren's syndrome
EP3318634A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations
US8609088B2 (en) 2011-05-10 2013-12-17 Regents Of The University Of Minnesota Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
EP3202914B2 (en) 2011-08-31 2023-06-28 The University of Manchester Method for treating a neurodegenerative disease
WO2013036833A1 (en) * 2011-09-09 2013-03-14 Mayo Foundation For Medical Education And Research Detecting frontotemporal dementia and amyotrophic lateral sclerosis
EP3170899B1 (en) * 2011-10-11 2020-06-24 The Brigham and Women's Hospital, Inc. Microrna mir-155 in neurodegenerative disorders
US20130142861A1 (en) 2011-12-05 2013-06-06 National Yang Ming University Compositions And Method For Detecting And Treating Abnormal Liver Homeostasis And Hepatocarcinogenesis
KR102057540B1 (ko) 2012-02-17 2019-12-19 더 칠드런스 호스피탈 오브 필라델피아 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법
EP2847337A4 (en) 2012-05-09 2016-04-27 Univ Oregon Health & Science ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS
EP3536795A3 (en) * 2012-06-21 2019-12-11 Association Institut de Myologie Widespread gene expression
KR20150070278A (ko) * 2012-10-15 2015-06-24 아이시스 파마수티컬즈 인코포레이티드 C9orf72 발현 조절용 조성물
EP2906697A4 (en) 2012-10-15 2016-06-22 Ionis Pharmaceuticals Inc METHOD FOR MONITORING THE C9ORF72 EXPRESSION
WO2014093622A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
AU2014205134B2 (en) 2013-01-14 2020-01-16 Recombinetics, Inc. Hornless livestock
WO2014114303A1 (en) 2013-01-22 2014-07-31 Deutsches Zentrum Für Neurodegenerative Erkrankungen Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion
CA2905952A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
CN110885854A (zh) 2013-03-15 2020-03-17 北卡罗来纳-查佩尔山大学 双重聚糖结合aav载体的方法和组合物
AU2014255665B2 (en) 2013-04-18 2018-08-02 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
US10006049B2 (en) 2013-05-16 2018-06-26 University Of Florida Research Foundation, Incorporated Hairpin mRNA elements and methods for the regulation of protein translation
JP7085716B2 (ja) 2013-06-05 2022-06-17 デューク ユニバーシティ Ｒｎａガイド遺伝子編集及び遺伝子調節
RU2716420C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка и применение систем crispr-cas, векторов и композиций для целенаправленного воздействия и терапии в печени
US10155794B2 (en) 2013-07-16 2018-12-18 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of disorders related to CEP290
CA2919995A1 (en) 2013-08-05 2015-02-12 Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp
SI3702466T1 (sl) * 2013-08-27 2023-04-28 Research Institute At Nationwide Children's Hospital Proizvodi in postopki za zdravljenje amiotrofične lateralne skleroze
EP3055414A4 (en) * 2013-10-11 2017-07-19 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
CA2932436A1 (en) 2013-12-12 2015-06-18 The Broad Institute, Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
GB201401707D0 (en) 2014-01-31 2014-03-19 Sec Dep For Health The Adeno-associated viral vectors
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
US10400227B2 (en) 2014-03-17 2019-09-03 The University Of Manitoba β-hexosaminidase protein variants and associated methods for treating GM2 gangliosidoses
EP3750907A3 (en) 2014-03-18 2021-04-28 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
EA034355B1 (ru) 2014-05-02 2020-01-30 Джензим Корпорейшн Векторы на основе aav для генной терапии в сетчатке и cns
RU2711147C2 (ru) 2014-05-20 2020-01-15 Юниверсити Оф Айова Рисерч Фаундейшн Терапевтические соединения для лечения болезни хантингтона
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
WO2015195621A1 (en) 2014-06-16 2015-12-23 The Johns Hopkins University Compositions and methods for the expression of crispr guide rnas using the h1 promoter
BR112017001725A2 (pt) 2014-07-31 2018-02-14 Association Inst De Myologie tratamento de esclerose lateral amiotrófica
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
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
US10407678B2 (en) 2015-04-16 2019-09-10 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
WO2016172155A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
WO2016174056A1 (en) 2015-04-27 2016-11-03 Genethon Compositions and methods for the treatment of nucleotide repeat expansion disorders
US10016514B2 (en) 2015-05-15 2018-07-10 New Hope Research Foundation Polynucleotides, vectors and methods for insertion and expression of transgenes
CA2986021A1 (en) 2015-05-16 2016-11-24 Genzyme Corporation Gene editing of deep intronic mutations
JP7002120B2 (ja) 2015-06-01 2022-01-20 ユニバーシティ オブ マサチューセッツ カテーテル組立体
WO2016210372A2 (en) 2015-06-25 2016-12-29 University Of Southern California Methods to treat neurological diseases
WO2017019876A1 (en) 2015-07-28 2017-02-02 University Of Massachusetts Transgenic expression of dnase i in vivo delivered by an adeno-associated virus vector
WO2017023724A1 (en) 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
US20210260219A1 (en) 2015-12-23 2021-08-26 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
WO2018064600A1 (en) 2016-09-30 2018-04-05 Regeneron Pharmaceuticals, Inc. Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus
CA3059213A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
AU2018338188B2 (en) 2017-09-22 2025-02-20 University Of Massachusetts SOD1 dual expression vectors and uses thereof

Also Published As

Publication number Publication date
EP3119797A4 (en) 2018-04-18
NZ724508A (en) 2024-05-31
JP2024032730A (ja) 2024-03-12
EP3750907A2 (en) 2020-12-16
EP3119797B1 (en) 2020-12-23
DK3119797T3 (da) 2021-03-15
AU2023200576A1 (en) 2023-04-27
US20190316126A1 (en) 2019-10-17
US10851375B2 (en) 2020-12-01
JP7051118B2 (ja) 2022-04-11
EP4410805A2 (en) 2024-08-07
JP2017510298A (ja) 2017-04-13
NZ762477A (en) 2024-05-31
US20240076668A1 (en) 2024-03-07
US20170114340A1 (en) 2017-04-27
US20190276826A1 (en) 2019-09-12
US20200032256A1 (en) 2020-01-30
JP2021152030A (ja) 2021-09-30
US10954518B2 (en) 2021-03-23
AU2015231294A1 (en) 2016-10-06
US11760999B2 (en) 2023-09-19
JP6756700B2 (ja) 2020-09-16
US20210246450A1 (en) 2021-08-12
WO2015143078A1 (en) 2015-09-24
EP3119797A1 (en) 2017-01-25
JP2020019772A (ja) 2020-02-06
AU2020260765B2 (en) 2022-12-01
CA2942515A1 (en) 2015-09-24
US10280418B2 (en) 2019-05-07
EP4410805A3 (en) 2024-11-27
AU2020260765A1 (en) 2020-11-26
AU2015231294B2 (en) 2020-10-29
EP3750907A3 (en) 2021-04-28
ES2856403T3 (es) 2021-09-27
US10711274B2 (en) 2020-07-14
AU2023200576B2 (en) 2024-08-22

Similar Documents

Publication Publication Date Title
AU2023200576B2 (en) Raav-based compositions and methods for treating amyotrophic lateral sclerosis
US11920133B2 (en) RAAV-based compositions and methods
JP7504967B2 (ja) ＭｅＣＰ２発現カセット
HK40115544A (en) Raav-based compositions and methods for treating amyotrophic lateral sclerosis
HK40041919A (en) Raav-based compositions and methods for treating amyotrophic lateral sclerosis
US20250319208A1 (en) Neuron specific promoters for aav gene transfer
HK1233649B (en) Raav-based compositions and methods for treating amyotrophic lateral sclerosis

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20200311

EEER Examination request

Effective date: 20200311

EEER Examination request

Effective date: 20200311

EEER Examination request

Effective date: 20200311

EEER Examination request

Effective date: 20200311

EEER Examination request

Effective date: 20200311