JP2019513779A5 - - Google Patents
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- JP2019513779A5 JP2019513779A5 JP2018553928A JP2018553928A JP2019513779A5 JP 2019513779 A5 JP2019513779 A5 JP 2019513779A5 JP 2018553928 A JP2018553928 A JP 2018553928A JP 2018553928 A JP2018553928 A JP 2018553928A JP 2019513779 A5 JP2019513779 A5 JP 2019513779A5
- Authority
- JP
- Japan
- Prior art keywords
- aav vector
- seq
- recombinant aav
- nucleotide sequence
- composition
- 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.)
- Granted
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- 239000013607 AAV vector Substances 0.000 claims description 65
- 239000000203 mixture Substances 0.000 claims description 55
- 239000002773 nucleotide Substances 0.000 claims description 50
- 125000003729 nucleotide group Chemical group 0.000 claims description 50
- 210000003205 muscle Anatomy 0.000 claims description 36
- 108091047189 miR-29c stem-loop Proteins 0.000 claims description 34
- 108091054490 miR-29c-2 stem-loop Proteins 0.000 claims description 34
- 102000001039 Dystrophin Human genes 0.000 claims description 21
- 108010069091 Dystrophin Proteins 0.000 claims description 21
- 201000006938 muscular dystrophy Diseases 0.000 claims description 21
- 206010016654 Fibrosis Diseases 0.000 claims description 15
- 230000004761 fibrosis Effects 0.000 claims description 15
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 10
- 239000003814 drug Substances 0.000 claims description 8
- 238000007911 parenteral administration Methods 0.000 claims description 7
- 238000007910 systemic administration Methods 0.000 claims description 7
- 238000010253 intravenous injection Methods 0.000 claims description 6
- 239000013598 vector Substances 0.000 claims description 6
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 5
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 5
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 5
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 5
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 5
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 5
- 241000649045 Adeno-associated virus 10 Species 0.000 claims description 5
- 241000649046 Adeno-associated virus 11 Species 0.000 claims description 5
- 241000649047 Adeno-associated virus 12 Species 0.000 claims description 5
- 241000300529 Adeno-associated virus 13 Species 0.000 claims description 5
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 5
- 230000007812 deficiency Effects 0.000 claims description 5
- 238000007918 intramuscular administration Methods 0.000 claims description 5
- 102000004169 proteins and genes Human genes 0.000 claims description 5
- 108090000623 proteins and genes Proteins 0.000 claims description 5
- 230000005856 abnormality Effects 0.000 claims description 4
- 239000003795 chemical substances by application Substances 0.000 claims description 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 4
- 208000035475 disorder Diseases 0.000 claims description 4
- 230000001105 regulatory effect Effects 0.000 claims description 4
- 230000003387 muscular Effects 0.000 claims description 3
- 238000010255 intramuscular injection Methods 0.000 claims description 2
- 239000007927 intramuscular injection Substances 0.000 claims description 2
- 230000001225 therapeutic effect Effects 0.000 claims description 2
- 238000000034 method Methods 0.000 description 11
- 108091007431 miR-29 Proteins 0.000 description 4
- 239000013608 rAAV vector Substances 0.000 description 4
- 108091029162 miR-29 stem-loop Proteins 0.000 description 2
- 238000002648 combination therapy Methods 0.000 description 1
- 229940079593 drug Drugs 0.000 description 1
- 230000004064 dysfunction Effects 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662323163P | 2016-04-15 | 2016-04-15 | |
| US62/323,163 | 2016-04-15 | ||
| US201762473253P | 2017-03-17 | 2017-03-17 | |
| US62/473,253 | 2017-03-17 | ||
| PCT/US2017/027635 WO2017181014A1 (en) | 2016-04-15 | 2017-04-14 | Adeno-associated virus vector delivery of microrna-29 and micro-dystrophin to treat muscular dystrophy |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019513779A JP2019513779A (ja) | 2019-05-30 |
| JP2019513779A5 true JP2019513779A5 (enExample) | 2020-05-28 |
| JP7189771B2 JP7189771B2 (ja) | 2022-12-14 |
Family
ID=60041940
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018553951A Active JP7078546B2 (ja) | 2016-04-15 | 2017-04-14 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2018553928A Active JP7189771B2 (ja) | 2016-04-15 | 2017-04-14 | 筋ジストロフィーを治療するためのマイクロｒｎａ－２９およびマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2018553882A Active JP7153562B2 (ja) | 2016-04-15 | 2017-04-14 | 筋ジストロフィーを治療するためのマイクロｒｎａ－２９のアデノ随伴ウイルスベクター送達 |
| JP2021190072A Active JP7431789B2 (ja) | 2016-04-15 | 2021-11-24 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2023201579A Active JP7833440B2 (ja) | 2016-04-15 | 2023-11-29 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2025075669A Pending JP2025118741A (ja) | 2016-04-15 | 2025-04-30 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018553951A Active JP7078546B2 (ja) | 2016-04-15 | 2017-04-14 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018553882A Active JP7153562B2 (ja) | 2016-04-15 | 2017-04-14 | 筋ジストロフィーを治療するためのマイクロｒｎａ－２９のアデノ随伴ウイルスベクター送達 |
| JP2021190072A Active JP7431789B2 (ja) | 2016-04-15 | 2021-11-24 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2023201579A Active JP7833440B2 (ja) | 2016-04-15 | 2023-11-29 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| JP2025075669A Pending JP2025118741A (ja) | 2016-04-15 | 2025-04-30 | 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
Country Status (26)
| Country | Link |
|---|---|
| US (7) | US11723986B2 (enExample) |
| EP (5) | EP3442561B1 (enExample) |
| JP (6) | JP7078546B2 (enExample) |
| KR (3) | KR20250061772A (enExample) |
| CN (2) | CN118895311A (enExample) |
| AU (4) | AU2017250790B2 (enExample) |
| CA (2) | CA3020996A1 (enExample) |
| CO (1) | CO2018012084A2 (enExample) |
| DK (1) | DK3442602T3 (enExample) |
| ES (3) | ES2977730T3 (enExample) |
| FI (1) | FI3442602T3 (enExample) |
| HR (1) | HRP20250541T1 (enExample) |
| HU (1) | HUE071243T2 (enExample) |
| IL (3) | IL319636A (enExample) |
| LT (1) | LT3442602T (enExample) |
| MA (2) | MA44683B1 (enExample) |
| MD (1) | MD3442602T2 (enExample) |
| MX (2) | MX2018012604A (enExample) |
| MY (2) | MY209041A (enExample) |
| PL (1) | PL3442602T3 (enExample) |
| PT (1) | PT3442602T (enExample) |
| RS (1) | RS66847B1 (enExample) |
| SG (1) | SG11201808941PA (enExample) |
| SI (1) | SI3442602T1 (enExample) |
| WO (3) | WO2017181011A1 (enExample) |
| ZA (2) | ZA201806746B (enExample) |
Families Citing this family (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3377042A4 (en) | 2015-11-16 | 2019-05-29 | The Research Institute at Nationwide Children's Hospital | MATERIALS AND METHOD FOR THE TREATMENT OF TITIN-BASED MYOPATHIES AND OTHER TITINOPATHIES |
| DK3442602T3 (da) | 2016-04-15 | 2025-05-19 | Res Inst Nationwide Childrens Hospital | Adeno-associeret virusvektors frigørelse af mikrodystrofin til behandling af muskeldystrofi |
| DK3596222T5 (da) * | 2017-03-17 | 2024-09-02 | Res Inst Nationwide Childrens Hospital | Adenoassocieret virusvektorfremføring af muskelspecifik mikro-dystrophin til behandling af muskeldystrofi |
| MA47800A (fr) | 2017-03-17 | 2020-01-22 | Univ Newcastle | Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire |
| WO2019078916A1 (en) * | 2017-10-18 | 2019-04-25 | Research Institute At Nationwide Children's Hospital | ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY |
| IL321310A (en) | 2017-10-20 | 2025-08-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for NT-3 gene therapy |
| AR114350A1 (es) | 2018-01-31 | 2020-08-26 | Res Institute At Nationwide ChildrenS Hospital | Terapia genética para la distrofia muscular de cinturas del tipo 2c |
| KR20250111393A (ko) * | 2018-03-16 | 2025-07-22 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달 |
| WO2019220435A1 (en) * | 2018-05-15 | 2019-11-21 | Ramot At Tel-Aviv University Ltd. | Mir126-5p for treating motor neuron diseases |
| US20240254181A1 (en) * | 2018-06-18 | 2024-08-01 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies |
| BR112020025995A2 (pt) * | 2018-06-18 | 2021-03-23 | Research Institute At Nationwide Children's Hospital | administração de microdistrofina músculo-específica por vetor de vírus adeno-associado para tratar a distrofia muscular |
| WO2020006458A1 (en) | 2018-06-29 | 2020-01-02 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| EP3598978B1 (en) | 2018-07-26 | 2024-05-29 | EXOFIX S.r.l. | Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles |
| KR20210124969A (ko) * | 2018-12-12 | 2021-10-15 | 솔리드 바이오사이언시즈 인크. | 근이영양증의 치료를 위한 조합 요법 |
| CA3131390A1 (en) | 2019-02-26 | 2020-09-03 | Research Institute Of Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
| WO2020193636A1 (en) * | 2019-03-25 | 2020-10-01 | Genethon | Production of large-sized quasidystrophins using overlapping aav vectors |
| EP4488287A3 (en) | 2019-08-21 | 2025-03-05 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
| EP4087617A4 (en) * | 2020-01-10 | 2024-05-01 | Solid Biosciences Inc. | VIRAL VECTOR FOR COMBINATION THERAPY |
| WO2021142435A1 (en) * | 2020-01-10 | 2021-07-15 | Solid Biosciences Inc. | Combination therapy for treating muscular dystrophy |
| TW202206447A (zh) | 2020-04-29 | 2022-02-16 | 美商必治妥美雅史谷比公司 | 具血影蛋白(spectrin)融合結構域之微型化肌肉萎縮蛋白及其用途 |
| BR112022025586A2 (pt) * | 2020-06-15 | 2023-03-07 | Res Inst Nationwide Childrens Hospital | Liberação de vetor de vírus adenoassociado para distrofias musculares |
| CN111733185A (zh) * | 2020-06-30 | 2020-10-02 | 华南农业大学 | 通过Mdfi基因编辑来实现动物肉品质改良的方法和应用 |
| EP3936616A1 (en) * | 2020-07-10 | 2022-01-12 | Charité - Universitätsmedizin Berlin | Microrna-targeted therapy for cardiac repair |
| US20240350674A1 (en) | 2021-08-11 | 2024-10-24 | Solid Biosciences Inc. | Treatment of muscular dystrophy |
| EP4401794A4 (en) * | 2021-09-16 | 2025-09-17 | Univ Florida | AAV particles with modified inverted terminal repeats for enhanced gene expression in muscle |
| US12285497B2 (en) | 2021-10-15 | 2025-04-29 | Research Institute At Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| US20250034590A1 (en) * | 2021-12-07 | 2025-01-30 | Biogen Ma Inc. | Methods and systems for transfecting host cells |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| CN115819546B (zh) * | 2022-08-19 | 2025-12-05 | 成都金唯科生物科技有限公司 | 表达微缩抗肌营养不良蛋白基因的腺相关病毒载体及其应用 |
| US20260055144A1 (en) | 2022-08-24 | 2026-02-26 | Regenxbio Inc. | Recombinant adeno-associated viruses and uses thereof |
| TW202603165A (zh) * | 2024-03-04 | 2026-01-16 | 美商凱特治療公司 | 用於治療杜興氏肌肉失養症之腺相關病毒組成物 |
| KR20250144954A (ko) | 2024-03-27 | 2025-10-13 | 주식회사 엘지에너지솔루션 | 양극 활물질, 이의 제조 방법, 이를 포함하는 양극 및 리튬 이차 전지 |
| WO2026011009A1 (en) * | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Compositions and methods for muscle disorders |
| WO2026075999A2 (en) | 2024-10-01 | 2026-04-09 | Sarepta Therapeutics, Inc. | Dystrophin and micro-dystrophin antibodies and fragments thereof |
Family Cites Families (54)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| ATE272123T1 (de) | 1993-11-09 | 2004-08-15 | Ohio Med College | Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved AAV vectors for gene therapy |
| US5795872A (en) | 1995-09-19 | 1998-08-18 | Pharmadigm, Inc. | DNA construct for immunization |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| AU723497C (en) | 1996-09-06 | 2001-10-11 | Trustees Of The University Of Pennsylvania, The | Method for recombinant adeno-associated virus-directed gene therapy |
| DK1944362T3 (en) | 1997-09-05 | 2016-01-25 | Genzyme Corp | Fremgangsmåder til fremstilling af hjælpevirusfri præparater med høj titer af rekombinante AAV-vektorer |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| JPH11318467A (ja) | 1998-05-08 | 1999-11-24 | Japan Science & Technology Corp | 短縮型ジストロフィン |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| AU2001255575B2 (en) | 2000-04-28 | 2006-08-31 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| EP1287125B1 (en) | 2000-04-28 | 2009-07-29 | Asklepios Biopharmaceutical, Inc. | Dna sequences encoding dystrophin minigenes and methods of use thereof |
| US20110097761A1 (en) | 2000-07-14 | 2011-04-28 | Chamberlain Jeffrey S | Mutant muscle-specific enhancers |
| US6869777B2 (en) | 2000-10-06 | 2005-03-22 | Regents Of The University Of Michigan | Mini-dystrophin nucleic acid sequences |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| ES2975413T3 (es) | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| CA2554818A1 (en) * | 2004-02-09 | 2005-08-25 | Thomas Jefferson University | Diagnosis and treatment of cancers with microrna located in or near cancer-associated chromosomal features |
| US20080044393A1 (en) | 2004-07-16 | 2008-02-21 | White Robert L | Retinal dystrophin transgene and methods of use thereof |
| US20100184209A1 (en) | 2006-02-17 | 2010-07-22 | Dharmacon, Inc. | Compositions and methods for inhibiting gene silencing by rna interference |
| US7892824B2 (en) | 2007-01-18 | 2011-02-22 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma |
| ES2621161T3 (es) | 2007-07-31 | 2017-07-03 | The Board Of Regents Of The University Of Texas System | Familia de micro-ARN que modula la fibrosis y usos de la misma |
| US8236557B2 (en) | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
| WO2010071454A1 (en) | 2008-12-17 | 2010-06-24 | Auckland Uniservices Limited | Adeno-associated viral vectors and uses thereof |
| AU2012286994B2 (en) | 2011-07-25 | 2017-08-10 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of DUX4 |
| WO2013075008A1 (en) * | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| US20150045416A1 (en) | 2012-01-05 | 2015-02-12 | Hadasit Medical Research Services & Development Ltd. | Methods and Compositions for Gene Delivery |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| US9624282B2 (en) | 2012-11-26 | 2017-04-18 | The Curators Of The University Of Missouri | Microdystrophin peptides and methods for treating muscular dystrophy using the same |
| EA201990558A3 (ru) * | 2013-04-20 | 2021-02-26 | Рисёрч Инститъют Эт Нэйшнвайд Чилдрен'С Хоспитал | Доставка нацеленных на экзон 2 полинуклеотидных конструкций u7snrna при помощи рекомбинантного аденоассоциированного вируса |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| GB201400598D0 (en) * | 2014-01-14 | 2014-03-05 | Univ Glasgow | Materials and methods for modulation of tendon healing |
| SG11201605906UA (en) | 2014-01-21 | 2016-08-30 | Univ Bruxelles | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
| US9439924B2 (en) | 2014-04-09 | 2016-09-13 | University Of Houston | Therapeutic miRNAs for treating heart and skeletal muscle diseases |
| WO2015161255A1 (en) | 2014-04-18 | 2015-10-22 | Board Of Regents Of The Nevada System Of Higher Education, On Behalf Of The University Of Nevada, Reno | Methods for enhancing or decreasing the levels of mir124 and mir29 in subjects with muscular dystrophy |
| EP2960336A1 (en) * | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
| EP3180435A4 (en) * | 2014-08-09 | 2018-01-17 | The Research Institute at Nationwide Children's Hospital | Methods and materials for activating an internal ribosomal entry site in exon 5 of the dmd gene |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| WO2016057975A2 (en) * | 2014-10-10 | 2016-04-14 | Research Institute At Nationwide Children's Hospital | Guided injections for aav gene transfer to muscle |
| JP6832280B2 (ja) | 2015-01-16 | 2021-02-24 | ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| GB201507842D0 (en) | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
| AU2016323575B2 (en) | 2015-09-17 | 2022-10-27 | Research Institute At Nationwide Children's Hospital | Methods and materials for GALGT2 gene therapy |
| WO2017165859A1 (en) | 2016-03-24 | 2017-09-28 | Research Institute At Nationwide Children's Hospital | Modified viral capsid proteins |
| DK3442602T3 (da) | 2016-04-15 | 2025-05-19 | Res Inst Nationwide Childrens Hospital | Adeno-associeret virusvektors frigørelse af mikrodystrofin til behandling af muskeldystrofi |
| EP4407035A3 (en) * | 2016-04-15 | 2024-10-30 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy |
| CA2971303C (en) | 2016-06-21 | 2026-03-03 | Bamboo Therapeutics, Inc. | Optimized mini-dystrophin genes and expression cassettes and their use |
| BR112020025995A2 (pt) | 2018-06-18 | 2021-03-23 | Research Institute At Nationwide Children's Hospital | administração de microdistrofina músculo-específica por vetor de vírus adeno-associado para tratar a distrofia muscular |
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2017
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