JP2019513399A5 - - Google Patents
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- Publication number
- JP2019513399A5 JP2019513399A5 JP2018554055A JP2018554055A JP2019513399A5 JP 2019513399 A5 JP2019513399 A5 JP 2019513399A5 JP 2018554055 A JP2018554055 A JP 2018554055A JP 2018554055 A JP2018554055 A JP 2018554055A JP 2019513399 A5 JP2019513399 A5 JP 2019513399A5
- Authority
- JP
- Japan
- Prior art keywords
- aav vector
- recombinant aav
- composition
- seq
- muscle
- 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
Links
- 239000013607 AAV vector Substances 0.000 claims 25
- 239000000203 mixture Substances 0.000 claims 18
- 210000003205 muscle Anatomy 0.000 claims 9
- 239000002773 nucleotide Substances 0.000 claims 8
- 125000003729 nucleotide group Chemical group 0.000 claims 8
- 108091033319 polynucleotide Proteins 0.000 claims 6
- 239000002157 polynucleotide Substances 0.000 claims 6
- 102000040430 polynucleotide Human genes 0.000 claims 6
- 108010059343 MM Form Creatine Kinase Proteins 0.000 claims 5
- 201000006938 muscular dystrophy Diseases 0.000 claims 5
- 208000034067 Beta-sarcoglycan-related limb-girdle muscular dystrophy R4 Diseases 0.000 claims 3
- 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 2
- 108010085238 Actins Proteins 0.000 claims 2
- 206010016654 Fibrosis Diseases 0.000 claims 2
- 108090000362 Lymphotoxin-beta Proteins 0.000 claims 2
- 102000005604 Myosin Heavy Chains Human genes 0.000 claims 2
- 108010084498 Myosin Heavy Chains Proteins 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 108091027981 Response element Proteins 0.000 claims 2
- 208000027418 Wounds and injury Diseases 0.000 claims 2
- 230000008602 contraction Effects 0.000 claims 2
- 230000006378 damage Effects 0.000 claims 2
- 230000004761 fibrosis Effects 0.000 claims 2
- 208000014674 injury Diseases 0.000 claims 2
- 238000010255 intramuscular injection Methods 0.000 claims 2
- 238000010253 intravenous injection Methods 0.000 claims 2
- 239000013598 vector Substances 0.000 claims 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 1
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 1
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 1
- 241000300529 Adeno-associated virus 13 Species 0.000 claims 1
- 102000019370 Beta-sarcoglycan Human genes 0.000 claims 1
- 102000004420 Creatine Kinase Human genes 0.000 claims 1
- 108010042126 Creatine kinase Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims 1
- 206010021143 Hypoxia Diseases 0.000 claims 1
- 201000009342 Limb-girdle muscular dystrophy Diseases 0.000 claims 1
- 108010083379 Sarcoglycans Proteins 0.000 claims 1
- 108010065729 Troponin I Proteins 0.000 claims 1
- 230000005856 abnormality Effects 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 201000009553 autosomal recessive limb-girdle muscular dystrophy type 2E Diseases 0.000 claims 1
- 230000000747 cardiac effect Effects 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 239000003862 glucocorticoid Substances 0.000 claims 1
- 230000007954 hypoxia Effects 0.000 claims 1
- 239000007943 implant Substances 0.000 claims 1
- 238000001802 infusion Methods 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 239000007927 intramuscular injection Substances 0.000 claims 1
- 108091047189 miR-29c stem-loop Proteins 0.000 claims 1
- 108091054490 miR-29c-2 stem-loop Proteins 0.000 claims 1
- 210000000663 muscle cell Anatomy 0.000 claims 1
- 238000007911 parenteral administration Methods 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 208000022561 qualitative or quantitative defects of beta-sarcoglycan Diseases 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 210000002027 skeletal muscle Anatomy 0.000 claims 1
- 150000003431 steroids Chemical class 0.000 claims 1
- 238000007910 systemic administration Methods 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022061771A JP7390426B2 (ja) | 2016-04-15 | 2022-04-01 | β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662323333P | 2016-04-15 | 2016-04-15 | |
| US62/323,333 | 2016-04-15 | ||
| US201662433548P | 2016-12-13 | 2016-12-13 | |
| US62/433,548 | 2016-12-13 | ||
| PCT/US2017/027583 WO2017180976A1 (en) | 2016-04-15 | 2017-04-14 | Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022061771A Division JP7390426B2 (ja) | 2016-04-15 | 2022-04-01 | β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019513399A JP2019513399A (ja) | 2019-05-30 |
| JP2019513399A5 true JP2019513399A5 (cg-RX-API-DMAC7.html) | 2020-05-14 |
| JP7079733B2 JP7079733B2 (ja) | 2022-06-02 |
Family
ID=60042261
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018554055A Active JP7079733B2 (ja) | 2016-04-15 | 2017-04-14 | β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
| JP2022061771A Active JP7390426B2 (ja) | 2016-04-15 | 2022-04-01 | β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022061771A Active JP7390426B2 (ja) | 2016-04-15 | 2022-04-01 | β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
Country Status (28)
Families Citing this family (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2841572B1 (en) | 2012-04-27 | 2019-06-19 | Duke University | Genetic correction of mutated genes |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| EP3020403A1 (en) | 2014-11-14 | 2016-05-18 | Universitat de Valéncia | Compounds for the treatment of myotonic dystrophy |
| US10676726B2 (en) | 2015-02-09 | 2020-06-09 | Duke University | Compositions and methods for epigenome editing |
| US11970710B2 (en) | 2015-10-13 | 2024-04-30 | Duke University | Genome engineering with Type I CRISPR systems in eukaryotic cells |
| 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 |
| EP3384055B1 (en) | 2015-11-30 | 2025-07-16 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| RS65490B1 (sr) * | 2016-04-15 | 2024-05-31 | Res Inst Nationwide Childrens Hospital | Vektor adeno-asociranog virusa za isporuku β-sarkoglikana i mikrorna-29 i tretman mišićne distrofije |
| MA45477A (fr) * | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
| US12214056B2 (en) | 2016-07-19 | 2025-02-04 | Duke University | Therapeutic applications of CPF1-based genome editing |
| JP7162021B2 (ja) | 2017-03-17 | 2022-10-27 | ニューカッスル ユニバーシティ | 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断片のアデノ随伴ウイルスベクター送達 |
| MY207563A (en) * | 2017-10-20 | 2025-03-04 | Res Inst Nationwide Childrens Hospital | Methods and materials for nt-3 gene therapy |
| TWI815856B (zh) | 2018-01-31 | 2023-09-21 | 美國全美兒童醫院之研究學會 | 用於肢帶型肌肉失養症2c型之基因療法 |
| PT3807413T (pt) | 2018-06-18 | 2025-08-19 | Res Inst Nationwide Childrens Hospital | Administração por vetor de vírus adenoassociado de microdistrofina específica do músculo para tratar a distrofia muscular |
| CN112543810A (zh) * | 2018-06-29 | 2021-03-23 | 全国儿童医院研究所 | 治疗肢带型肌营养不良2a型的重组腺相关病毒产品和方法 |
| EP3598978B1 (en) | 2018-07-26 | 2024-05-29 | EXOFIX S.r.l. | Fibroadipogenic progenitor-derived exosomes for regeneration of dystrophic muscles |
| US20220119489A1 (en) * | 2019-01-25 | 2022-04-21 | Children's Medical Center Corporation | Compositions and methods for treating muscular dystrophy and related disorders |
| JP2022523038A (ja) * | 2019-02-02 | 2022-04-21 | ユニバーシティ オブ ピッツバーグ －オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション | 遺伝子置換及び抗炎症を介したデュシェンヌ型筋ジストロフィーに対する一段階遺伝子療法 |
| BR112021016570A2 (pt) * | 2019-02-26 | 2021-11-03 | Res Inst Nationwide Childrens Hospital | Entrega de vetor de vírus adenoassociado de beta-sarcoglicano e tratamento de distrofia muscular |
| EP4374880A3 (en) | 2019-08-21 | 2024-08-14 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
| US20230040544A1 (en) * | 2019-12-16 | 2023-02-09 | Research Institute At Nationwide Children's Hospital | Compositions and methods for restoring and maintaining the dystrophin-associated protein complex (dapc) |
| JP2023530171A (ja) * | 2020-06-19 | 2023-07-13 | ジェネトン | 筋肉内及び心臓内でのｓｇｃｇの適切な発現を可能にする遺伝子療法発現系 |
| 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 |
| CA3260237A1 (en) | 2022-06-24 | 2023-12-28 | Tune Therapeutics, Inc. | Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression |
| WO2025132132A1 (en) | 2023-12-18 | 2025-06-26 | F. Hoffmann-La Roche Ag | Skeletal muscle-specific promoters |
| CN118045206B (zh) * | 2024-04-12 | 2024-07-05 | 四川至善唯新生物科技有限公司 | 一种治疗脊髓型肌肉萎缩的药物组合物及其用途 |
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| ES2216005T3 (es) | 1993-11-09 | 2004-10-16 | Targeted Genetics Corporation | Produccion de titulos elevados de vectores de aav recombinantes. |
| CA2176215C (en) | 1993-11-09 | 2007-06-26 | James P. Trempe | Stable cell lines capable of expressing the adeno-associated virus replication gene |
| 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 |
| EP0796339A1 (en) | 1994-12-06 | 1997-09-24 | 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 |
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| WO2001083692A2 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
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| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| RS61985B1 (sr) * | 2012-09-06 | 2021-07-30 | Univ Chicago | Antisens polinukleotidi za indukovanje preskakanja egzona i postupci lečenja distrofija |
| 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 |
| FR3004463A1 (fr) * | 2013-04-11 | 2014-10-17 | Genethon | Systeme d'expression pour une therapie genique selective |
| 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 |
| EP3800261A1 (en) | 2014-01-21 | 2021-04-07 | Vrije Universiteit Brussel | 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 |
| EP2960336A1 (en) | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
| 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 |
| RS65490B1 (sr) * | 2016-04-15 | 2024-05-31 | Res Inst Nationwide Childrens Hospital | Vektor adeno-asociranog virusa za isporuku β-sarkoglikana i mikrorna-29 i tretman mišićne distrofije |
| WO2021257595A1 (en) * | 2020-06-15 | 2021-12-23 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery for muscular dystrophies |
-
2017
- 2017-04-14 RS RS20240504A patent/RS65490B1/sr unknown
- 2017-04-14 CA CA3021057A patent/CA3021057A1/en active Pending
- 2017-04-14 IL IL262262A patent/IL262262B2/en unknown
- 2017-04-14 MX MX2018012605A patent/MX391208B/es unknown
- 2017-04-14 BR BR112018071167A patent/BR112018071167A2/pt unknown
- 2017-04-14 LT LTEPPCT/US2017/027583T patent/LT3442600T/lt unknown
- 2017-04-14 AU AU2017250756A patent/AU2017250756B2/en active Active
- 2017-04-14 PT PT177832078T patent/PT3442600T/pt unknown
- 2017-04-14 EP EP17783207.8A patent/EP3442600B1/en active Active
- 2017-04-14 MA MA044681A patent/MA44681A/fr unknown
- 2017-04-14 HR HRP20240580TT patent/HRP20240580T1/hr unknown
- 2017-04-14 ES ES17783207T patent/ES2979174T3/es active Active
- 2017-04-14 EP EP24163078.9A patent/EP4407035A3/en active Pending
- 2017-04-14 SG SG11201809029RA patent/SG11201809029RA/en unknown
- 2017-04-14 DK DK17783207.8T patent/DK3442600T5/da active
- 2017-04-14 HU HUE17783207A patent/HUE066442T2/hu unknown
- 2017-04-14 US US16/093,027 patent/US11358993B2/en active Active
- 2017-04-14 PL PL17783207.8T patent/PL3442600T3/pl unknown
- 2017-04-14 SI SI201731512T patent/SI3442600T1/sl unknown
- 2017-04-14 FI FIEP17783207.8T patent/FI3442600T3/fi active
- 2017-04-14 CN CN202210977746.2A patent/CN117384964A/zh active Pending
- 2017-04-14 CN CN201780023705.7A patent/CN109121395B/zh active Active
- 2017-04-14 JP JP2018554055A patent/JP7079733B2/ja active Active
- 2017-04-14 WO PCT/US2017/027583 patent/WO2017180976A1/en not_active Ceased
- 2017-04-14 SM SM20240169T patent/SMT202400169T1/it unknown
- 2017-04-14 MY MYPI2018001743A patent/MY195439A/en unknown
- 2017-06-20 KR KR1020187032688A patent/KR102502091B1/ko active Active
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2018
- 2018-10-15 ZA ZA2018/06863A patent/ZA201806863B/en unknown
- 2018-10-15 SA SA518400245A patent/SA518400245B1/ar unknown
- 2018-10-15 MX MX2022003857A patent/MX2022003857A/es unknown
- 2018-11-08 CO CONC2018/0012082A patent/CO2018012082A2/es unknown
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2021
- 2021-05-13 AU AU2021203044A patent/AU2021203044B2/en active Active
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2022
- 2022-01-20 US US17/580,228 patent/US20220251156A1/en not_active Abandoned
- 2022-04-01 JP JP2022061771A patent/JP7390426B2/ja active Active
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2023
- 2023-10-06 US US18/482,526 patent/US20240182533A1/en active Pending