JP2024523707A - 遺伝子治療のための最適化された発現カセット - Google Patents

遺伝子治療のための最適化された発現カセット Download PDF

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JP2024523707A
JP2024523707A JP2024500231A JP2024500231A JP2024523707A JP 2024523707 A JP2024523707 A JP 2024523707A JP 2024500231 A JP2024500231 A JP 2024500231A JP 2024500231 A JP2024500231 A JP 2024500231A JP 2024523707 A JP2024523707 A JP 2024523707A
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transgene
promoter
seq
expression cassette
enhancer
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JP2024523707A5 (enExample
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ファンユー ジョウ，
クリストファー エー． リード，
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テナヤ セラピューティクス， インコーポレイテッド
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    • 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
    • A61K48/005—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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C07K—PEPTIDES
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    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
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  • Zoology (AREA)
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  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Plant Pathology (AREA)
  • Virology (AREA)
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  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
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  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2024500231A 2021-07-08 2022-07-08 遺伝子治療のための最適化された発現カセット Pending JP2024523707A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163219651P 2021-07-08 2021-07-08
US63/219,651 2021-07-08
PCT/US2022/073574 WO2023283649A1 (en) 2021-07-08 2022-07-08 Optimized expression cassettes for gene therapy

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JP2024523707A true JP2024523707A (ja) 2024-06-28
JP2024523707A5 JP2024523707A5 (enExample) 2025-07-16

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US (2) US12151001B2 (enExample)
EP (1) EP4367251A1 (enExample)
JP (1) JP2024523707A (enExample)
KR (1) KR20240032971A (enExample)
AR (1) AR126407A1 (enExample)
AU (1) AU2022307365A1 (enExample)
CA (1) CA3224488A1 (enExample)
CO (1) CO2024001148A2 (enExample)
IL (1) IL309742A (enExample)
MX (1) MX2024000175A (enExample)
TW (1) TW202317768A (enExample)
WO (1) WO2023283649A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021142267A1 (en) 2020-01-08 2021-07-15 Scarpmap Llc Methods and computing system for processing ultrasound image to determine health of subdermal tissue
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
TW202317768A (zh) 2021-07-08 2023-05-01 美商特納亞治療股份有限公司 用於基因療法的優化表現卡匣
CA3240080A1 (en) * 2021-12-08 2023-06-15 Christopher Dean HERZOG Junctophilin-2 (jph2) gene therapy using aav vector
KR20250169529A (ko) * 2023-02-13 2025-12-03 아스트라제네카 아일랜드 리미티드 유전자 요법을 위한 Bcl2-관련 아타노진 3(BAG3)을 인코딩하는 핵산
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
EP4695396A1 (en) 2023-04-10 2026-02-18 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
AU2024294136A1 (en) * 2023-07-20 2026-02-05 Children's Medical Research Institute Modified cardiotropic aav capsid polypeptides and vectors
WO2025162449A1 (en) * 2024-01-30 2025-08-07 Inspirar Limited Compositions and methods for the treatment of heart disease
WO2025171086A1 (en) * 2024-02-07 2025-08-14 Asimov Inc. Promoters and uses thereof
WO2026099582A1 (en) * 2024-11-08 2026-05-15 Oxford University Innovation Limited Constructs for rna expression

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003506094A (ja) * 1999-08-06 2003-02-18 アンスティテュ パストゥール 心臓における遺伝子発現を活性化するポリヌクレオチドおよび遺伝子治療におけるその使用
JP2016516423A (ja) * 2013-04-17 2016-06-09 ウニヴェルジテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 心筋症を処置するための遺伝子治療ベクター
US20170298107A1 (en) * 2016-04-19 2017-10-19 The Board Of Regents Of The University Of Texas System Use of a small native peptide activator of serca pump for treatment of heart failure and other disorders characterized by cytosolic calcium overload
WO2020014523A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
WO2020047467A2 (en) * 2018-08-30 2020-03-05 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with myocardin and ascl1

Family Cites Families (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
JP2002511259A (ja) 1998-04-09 2002-04-16 ジェンセット ５’ｅｓｔおよびコードされるヒトタンパク質
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
DE69936104T2 (de) 1998-11-05 2008-01-17 The Trustees Of The University Of Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
US20020077470A1 (en) 1999-04-26 2002-06-20 Walker Michael G. Cardiac muscle-associated genes
AU7061401A (en) * 2000-07-05 2002-01-14 Transgene Sa Chimeric promoters for controlling expression in smooth muscle cells
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
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
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
DK2623605T3 (en) 2007-04-09 2019-03-25 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
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
EP2494058B1 (en) 2009-10-29 2017-04-05 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
PT2945642T (pt) * 2013-01-17 2024-05-27 Medizinische Hochschule Hannover Proteína do fator 1 para utilização no tratamento ou prevenção de doenças
EP3043866A4 (en) 2013-09-12 2017-05-17 President and Fellows of Harvard College Methods for inducing glucose uptake
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
US11124546B2 (en) 2015-12-14 2021-09-21 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
WO2017192750A1 (en) 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
JP7100629B2 (ja) 2016-06-09 2022-07-13 セントレ・ナショナル・デ・ラ・レシェルシェ・サイエンティフィーク 化学修飾されたカプシドを有するｒａａｖ
WO2018026766A1 (en) 2016-08-01 2018-02-08 The Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Compositions and methods for treating cardiac injury
EP3583220A4 (en) 2017-02-15 2021-07-07 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR GENERAL TRANSFER IN THE VASCULAR SYSTEM
CA3052705A1 (en) 2017-02-24 2018-08-30 The Board Of Regents Of The University Of Texas System Compositions and methods relating to myomixer-promoted muscle cell fusion
KR102616820B1 (ko) * 2017-03-17 2023-12-21 애드베룸 바이오테크놀로지스, 인코포레이티드 향상된 유전자 발현을 위한 조성물 및 방법
WO2018222503A1 (en) 2017-05-31 2018-12-06 The Regents Of The University Of California Adeno-associated virus with variant capsid and methods of use thereof
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
GB2571704A (en) * 2018-02-22 2019-09-11 Georg August Univ Gottingen Stiftung Offentlichen Rechts Univer Sitatsmedzin Goettingen Prevention or treatment of cardiac arrhytmia and sudden cardiac death
BR112021009913A2 (pt) 2018-12-05 2021-08-17 Abeona Therapeutics, Inc. vetor viral adeno-associado recombinante para inserção gênica
JP2022516515A (ja) * 2018-12-31 2022-02-28 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 Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
JP7659271B2 (ja) 2019-03-28 2025-04-09 ザ ジェネラル ホスピタル コーポレイション 導入遺伝子発現のための操作されたアデノ随伴（ａａｖ）ベクター
WO2021142267A1 (en) 2020-01-08 2021-07-15 Scarpmap Llc Methods and computing system for processing ultrasound image to determine health of subdermal tissue
FI4103214T3 (fi) * 2020-02-13 2026-07-03 Tenaya Therapeutics Inc Geeniterapian vektorit sydänsairauden hoitoon
US20230241249A1 (en) 2020-07-07 2023-08-03 The Board Of Regents Of The University Of Texas System Adeno-associated virus vector for dwarf open reading frame
WO2022032226A1 (en) * 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
TW202317768A (zh) 2021-07-08 2023-05-01 美商特納亞治療股份有限公司 用於基因療法的優化表現卡匣

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003506094A (ja) * 1999-08-06 2003-02-18 アンスティテュ パストゥール 心臓における遺伝子発現を活性化するポリヌクレオチドおよび遺伝子治療におけるその使用
JP2016516423A (ja) * 2013-04-17 2016-06-09 ウニヴェルジテーツクリニクム ハンブルク−エッペンドルフＵｎｉｖｅｒｓｉｔａｅｔｓｋｌｉｎｉｋｕｍ Ｈａｍｂｕｒｇ−Ｅｐｐｅｎｄｏｒｆ 心筋症を処置するための遺伝子治療ベクター
US20170298107A1 (en) * 2016-04-19 2017-10-19 The Board Of Regents Of The University Of Texas System Use of a small native peptide activator of serca pump for treatment of heart failure and other disorders characterized by cytosolic calcium overload
WO2020014523A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
WO2020047467A2 (en) * 2018-08-30 2020-03-05 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with myocardin and ascl1

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