JP2022551986A5 - - Google Patents

Info

Publication number
JP2022551986A5
JP2022551986A5 JP2022522782A JP2022522782A JP2022551986A5 JP 2022551986 A5 JP2022551986 A5 JP 2022551986A5 JP 2022522782 A JP2022522782 A JP 2022522782A JP 2022522782 A JP2022522782 A JP 2022522782A JP 2022551986 A5 JP2022551986 A5 JP 2022551986A5
Authority
JP
Japan
Prior art keywords
aforementioned
polypeptide
disease
vector system
aav
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.)
Pending
Application number
JP2022522782A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022551986A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/056133 external-priority patent/WO2021077000A1/en
Publication of JP2022551986A publication Critical patent/JP2022551986A/ja
Publication of JP2022551986A5 publication Critical patent/JP2022551986A5/ja
Priority to JP2025234566A priority Critical patent/JP2026070504A/ja
Pending legal-status Critical Current

Links

JP2022522782A 2019-10-16 2020-10-16 修飾筋肉標的化組成物 Pending JP2022551986A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025234566A JP2026070504A (ja) 2019-10-16 2025-12-05 修飾筋肉標的化組成物

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962916221P 2019-10-16 2019-10-16
US201962916207P 2019-10-16 2019-10-16
US62/916,221 2019-10-16
US62/916,207 2019-10-16
US202063018454P 2020-04-30 2020-04-30
US63/018,454 2020-04-30
US202063055252P 2020-07-22 2020-07-22
US63/055,252 2020-07-22
PCT/US2020/056133 WO2021077000A1 (en) 2019-10-16 2020-10-16 Engineered muscle targeting compositions

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025234566A Division JP2026070504A (ja) 2019-10-16 2025-12-05 修飾筋肉標的化組成物

Publications (2)

Publication Number Publication Date
JP2022551986A JP2022551986A (ja) 2022-12-14
JP2022551986A5 true JP2022551986A5 (enExample) 2023-10-18

Family

ID=75538392

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022522782A Pending JP2022551986A (ja) 2019-10-16 2020-10-16 修飾筋肉標的化組成物
JP2025234566A Pending JP2026070504A (ja) 2019-10-16 2025-12-05 修飾筋肉標的化組成物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025234566A Pending JP2026070504A (ja) 2019-10-16 2025-12-05 修飾筋肉標的化組成物

Country Status (7)

Country Link
US (5) US20220340929A1 (enExample)
EP (1) EP4045637A4 (enExample)
JP (2) JP2022551986A (enExample)
CN (1) CN115175991A (enExample)
AU (1) AU2020368539A1 (enExample)
CA (1) CA3153902A1 (enExample)
WO (1) WO2021077000A1 (enExample)

Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20220325296A1 (en) * 2019-09-12 2022-10-13 The Broad Institute, Inc. Engineered adeno-associated virus capsids
AU2021259422A1 (en) 2020-04-20 2022-12-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
MX2023005446A (es) 2020-11-11 2023-07-18 European Molecular Biology Laboratory Particulas virales modificadas para terapia genica.
AU2022262771A1 (en) * 2021-04-23 2023-11-02 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
WO2022226375A1 (en) * 2021-04-23 2022-10-27 Locanabio, Inc. Tissue-targeted modified aav capsids and methods of use thereof
CA3216419A1 (en) * 2021-04-23 2022-10-27 Greg NACHTRAB Tissue-targeted modified aav capsids and methods of use thereof
AU2022313258A1 (en) * 2021-07-19 2024-02-08 New York University Auf1 combination therapies for treatment of muscle degenerative disease
WO2023015304A1 (en) * 2021-08-05 2023-02-09 Insmed Incorporated Adeno-associated virus particles and methods of use thereof
WO2023015297A1 (en) * 2021-08-05 2023-02-09 The Broad Institute, Inc. Engineered muscle targeting compositions
EP4392039A4 (en) * 2021-08-24 2025-10-15 Prime Medicine Inc GENOME EDITING COMPOSITIONS AND METHODS FOR TREATING RETINOPATHY
EP4399316A4 (en) * 2021-09-08 2025-11-12 Broad Inst Inc MODIFIED FORMS FOR TARGETING THE CENTRAL NERVOUS SYSTEM AND MUSCLES
JP2025500347A (ja) * 2021-10-05 2025-01-09 ザ・ブロード・インスティテュート・インコーポレイテッド 操作された心筋組成物
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
WO2023111351A1 (en) * 2021-12-17 2023-06-22 Christian Kupatt Aav vectors for gene therapy in endothelial cells
EP4473001A1 (en) * 2022-02-01 2024-12-11 The Broad Institute, Inc. Adeno-associated viral vectors and uses thereof
WO2023183860A1 (en) * 2022-03-23 2023-09-28 University Of Delaware Engineered extracellular vesicles for targeted drug delivery to muscle
WO2024130070A2 (en) * 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
WO2024184781A1 (en) 2023-03-03 2024-09-12 Universitat Heidelberg Engineered aav capsids
JP2026509271A (ja) 2023-03-10 2026-03-17 ダイノ セラピューティクス,インコーポレーテッド カプシドポリペプチドおよびその使用方法
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
CN118955652A (zh) * 2023-05-15 2024-11-15 杭州新昶基因技术有限公司 腺相关病毒衣壳蛋白、包含其的腺相关病毒及应用
AU2024271851A1 (en) 2023-05-16 2025-12-18 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
EP4766844A1 (en) * 2023-08-25 2026-07-01 Agency for Science, Technology And Research COMPOSITION AND METHODS FOR IDENTIFYING NEW AAV VARIANTS FOR ADMINISTRATING DIFFERENTIATED GENES TO CARDIOMYOCTS AND/OR SKELETAL MYOFIBERS
KR20260082361A (ko) 2023-08-31 2026-06-04 디노 테라퓨틱스, 인코포레이티드 캡시드 폴리펩타이드 및 이의 사용 방법
CN116870198B (zh) * 2023-09-06 2023-11-28 中国农业大学 一种调节骨骼肌损伤修复的方法
WO2025054256A1 (en) * 2023-09-06 2025-03-13 Tenaya Therapeutics, Inc. Targeting peptides, engineered aav capsid proteins and virions
WO2025079110A1 (en) * 2023-10-11 2025-04-17 Peptris Technologies Private Limited Atovaquone for the treatment and management of muscular dystrophies
KR20260114566A (ko) * 2023-11-14 2026-07-22 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심근 및 골격근-특이적 표적화 모티프를 갖는 조성물 및 이의 용도
WO2025130904A1 (zh) * 2023-12-21 2025-06-26 中山大学中山眼科中心 Crb1基因在治疗眼部和肠道等全身疾病中的用途
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025185625A1 (zh) * 2024-03-04 2025-09-12 苏州新芽基因生物技术有限公司 特异性靶向人组织的多肽序列及其在构建递送载体中的应用
WO2025188755A1 (en) 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025217586A1 (en) * 2024-04-11 2025-10-16 The Children's Medical Center Corporation Compositions and methods for treating selenoprotein-associated diseases
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
TW202613313A (zh) 2024-05-31 2026-04-01 美商薩羅塔治療公司 肌向性raav
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2026011008A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Expression control by skeletal muscle-expressed mirnas
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
WO2026030244A1 (en) 2024-07-30 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a
WO2026050402A1 (en) 2024-08-29 2026-03-05 Kate Therapeutics, Inc. Liver de-targeted muscle tropic capsids
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026107393A2 (en) 2024-11-14 2026-05-21 The Broad Institute, Inc. Aav capsid modifications that reduce immunogenicity
WO2026122963A1 (en) 2024-12-06 2026-06-11 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026150324A1 (en) 2025-01-10 2026-07-16 Sarepta Therapeutics, Inc. Compositions and methods for treating dysferlinopathies
CN121344093B (zh) * 2025-11-26 2026-06-30 中国农业科学院北京畜牧兽医研究所 一种肌酸激酶启动子定点替换华西牛基因启动子的方法

Family Cites Families (80)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
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
US5846946A (en) 1996-06-14 1998-12-08 Pasteur Merieux Serums Et Vaccins Compositions and methods for administering Borrelia DNA
EP1108047A1 (en) 1998-08-27 2001-06-20 Aventis Pharma S.A. Targeted adenovirus vectors for delivery of heterologous genes
DE19933719A1 (de) 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
EP1268774A2 (en) 2000-03-21 2003-01-02 Elitra Pharmaceuticals, Inc. Identification of essential genes in prokaryotes
WO2002053703A2 (en) 2001-01-05 2002-07-11 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
US20040142325A1 (en) * 2001-09-14 2004-07-22 Liat Mintz Methods and systems for annotating biomolecular sequences
US20050106558A1 (en) * 2001-12-21 2005-05-19 Luca Perabo Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
WO2005075506A1 (en) * 2004-01-09 2005-08-18 The Scripps Research Institute Identification of endogenous trimerization domains in the adenovirus fiber protein that allow detargeting and retargeting of viral vectors
EP2397490B1 (en) 2004-07-16 2013-09-04 THE UNITED STATES OF AMERICA, represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Vaccine constructs and combinations of vaccines designed to improve the breadth of the immune response to diverse strains and clades of HIV
US8129352B2 (en) 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
WO2007093836A1 (en) 2006-02-13 2007-08-23 Cellectis Meganuclease variants cleaving a dna target sequence from a xp gene and uses thereof
WO2008010009A1 (en) 2006-07-18 2008-01-24 Cellectis Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof
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
WO2009013559A1 (en) 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
WO2009019528A1 (en) 2007-08-03 2009-02-12 Cellectis Meganuclease variants cleaving a dna target sequence from the human interleukin-2 receptor gamma chain gene and uses thereof
JP2011512326A (ja) 2007-12-31 2011-04-21 ナノコア セラピューティクス,インコーポレイテッド 心不全の治療用のrna干渉
US9273296B2 (en) 2008-09-08 2016-03-01 Cellectis Meganuclease variants cleaving a DNA target sequence from a glutamine synthetase gene and uses thereof
ES2738980T3 (es) 2008-09-15 2020-01-28 Childrens Medical Ct Corp Modulación de BCL11A para el tratamiento de hemoglobinopatías
US20110016540A1 (en) 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
US20110023145A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in autism spectrum disorders
US8592386B2 (en) 2008-12-17 2013-11-26 Sarepta Therapeutics, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
WO2010136549A2 (en) 2009-05-28 2010-12-02 Deutsches Krebsforschungszentrum Modified aav capsid polypeptides
EP2569424A1 (en) 2010-05-12 2013-03-20 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
US8198429B2 (en) 2010-08-09 2012-06-12 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
WO2012106725A2 (en) 2011-02-04 2012-08-09 Sangamo Biosciences, Inc. Methods and compositions for treating occular disorders
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
WO2013082548A1 (en) 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
WO2013086444A2 (en) 2011-12-08 2013-06-13 Sarepta Therapeutics, Inc. Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human lmna
EP2836226B1 (en) 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
CA2865011C (en) 2012-02-29 2021-06-15 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
EP3470523A1 (en) * 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP3760720A1 (en) 2013-03-14 2021-01-06 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
KR20160097338A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 반복 장애에서의 crispr-cas 시스템의 조성물 및 방법 및 용도
WO2015116568A2 (en) * 2014-01-28 2015-08-06 University Of Miami Muscle cell-targeting nanoparticles for vaccination and nucleic acid delivery, and methods of production and use thereof
US11028388B2 (en) 2014-03-05 2021-06-08 Editas Medicine, Inc. CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
CA2943622A1 (en) 2014-03-25 2015-10-01 Editas Medicine Inc. Crispr/cas-related methods and compositions for treating hiv infection and aids
EP3981876A1 (en) 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
WO2015148860A1 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
WO2015153791A1 (en) 2014-04-01 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus type 2 (hsv-2)
WO2015153789A1 (en) 2014-04-01 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating herpes simplex virus type 1 (hsv-1)
US10577627B2 (en) * 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
WO2016115543A2 (en) 2015-01-16 2016-07-21 University Of Washington Novel micro-dystrophins and related methods of use
MX2017011004A (es) 2015-02-27 2018-02-09 Sarepta Therapeutics Inc Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida.
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California アデノ随伴ウイルス変異体及びその使用方法
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
EP3307891B8 (en) * 2015-06-10 2019-11-13 Association Institut de Myologie Combined therapy for duchenne muscular dystrophy
US10544105B2 (en) 2015-07-08 2020-01-28 Cv6 Therapeutics (Ni) Limited Deoxyuridine triphosphatase inhibitors containing cyclopropano linkage
WO2017040271A1 (en) 2015-08-28 2017-03-09 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
US20190054113A1 (en) 2015-09-30 2019-02-21 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
JP2018530560A (ja) 2015-10-09 2018-10-18 サレプタ セラピューティクス, インコーポレイテッド デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法
WO2017096164A1 (en) 2015-12-02 2017-06-08 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
EA201891419A1 (ru) 2015-12-15 2019-01-31 Сарепта Терапьютикс, Инк. Пептид-олигонуклеотидные конъюгаты
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
US11060089B2 (en) 2016-04-18 2021-07-13 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
WO2017197355A2 (en) 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
US20230048025A1 (en) 2016-12-22 2023-02-16 Oregon Health & Science University Adeno associated viral vectors
US10055384B2 (en) 2016-12-31 2018-08-21 Entefy Inc. Advanced zero-knowledge document processing and synchronization
EP4219806A3 (en) 2017-04-11 2023-09-27 Ruprecht-Karls-Universität Heidelberg Adeno-associated virus library
US11771662B2 (en) 2017-09-05 2023-10-03 Kansas State University Research Foundation Analgesic formulation for control of pain in dogs
DK3684423T5 (da) * 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
TW201936201A (zh) 2017-12-14 2019-09-16 美商堅固生物科技公司 基因之非病毒生產及遞送
JP7374119B2 (ja) * 2018-04-05 2023-11-06 ジェネトン 減少した肝臓向性を有するAAV9とAAVrh74とのハイブリッド組換えアデノ随伴ウイルス血清型
CA3097375A1 (en) * 2018-04-27 2019-10-31 Universitat Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
IL278598B2 (en) 2018-05-11 2025-11-01 Massachusetts Eye & Ear Infirmary Altering tissue tropism of adeno-associated viruses
US20220096574A1 (en) 2019-01-29 2022-03-31 Holobiome, Inc. Methods and compositions for treating and preventing central nervous system disorders and other conditions caused by gut microbial dysbiosis
CN109897831B (zh) 2019-03-14 2021-05-28 苏州吉脉基因药物生物科技有限公司 具有突变衣壳的腺相关病毒病毒体及其应用
US20220325296A1 (en) * 2019-09-12 2022-10-13 The Broad Institute, Inc. Engineered adeno-associated virus capsids
IL291944A (en) * 2019-10-10 2022-06-01 Solid Biosciences Inc Modified aav capsids and uses thereof

Similar Documents

Publication Publication Date Title
JP2023534999A5 (enExample)
JP2020513811A5 (enExample)
JP2024099770A5 (enExample)
CN112004925B (zh) 具有降低的肝向性的AAV9和AAVrh74的杂合重组腺相关病毒血清型
JP2025162557A5 (enExample)
JP6174318B2 (ja) アデノ随伴ウイルス構築物を含んでなるコンダクティング気道細胞を標的とするための組成物
JP2024530023A5 (enExample)
JP2021512649A5 (enExample)
JP2020528734A5 (enExample)
JP2021512072A5 (enExample)
JP2024514956A5 (enExample)
IL273261B2 (en) Adeno-associated virus variant capsids and methods of using them
JP2022508182A5 (enExample)
JP2023514242A5 (enExample)
CN116096431A (zh) 肽修饰的aav衣壳
JP2025500347A5 (enExample)
JP7541027B2 (ja) 筋肉発現のためのハイブリッドプロモーター
JP2020510447A5 (enExample)
JP2022535749A5 (enExample)
JP2021520837A5 (enExample)
JP2023537358A5 (enExample)
JP2022547197A5 (enExample)
JP2023534037A5 (enExample)
JP2022551744A5 (enExample)
EP4536686B1 (en) Peptide-modified aav capsid with enhanced muscle transduction efficiency