ZA201806863B - Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy - Google Patents

Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy

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Publication number
ZA201806863B
ZA201806863B ZA2018/06863A ZA201806863A ZA201806863B ZA 201806863 B ZA201806863 B ZA 201806863B ZA 2018/06863 A ZA2018/06863 A ZA 2018/06863A ZA 201806863 A ZA201806863 A ZA 201806863A ZA 201806863 B ZA201806863 B ZA 201806863B
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ZA
South Africa
Prior art keywords
sarcoglycan
microrna
adeno
treatment
associated virus
Prior art date
Application number
ZA2018/06863A
Other languages
English (en)
Inventor
Rodino-Klapac Louise
R Mendell Jerry
Original Assignee
Res Inst Nationwide Childrens Hospital
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 Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of ZA201806863B publication Critical patent/ZA201806863B/en

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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
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    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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ZA2018/06863A 2016-04-15 2018-10-15 Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy ZA201806863B (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662323333P 2016-04-15 2016-04-15
US201662433548P 2016-12-13 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

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Publication Number Publication Date
ZA201806863B true ZA201806863B (en) 2019-12-18

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ZA2018/06863A ZA201806863B (en) 2016-04-15 2018-10-15 Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy

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US (3) US11358993B2 (enExample)
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AU (2) AU2017250756B2 (enExample)
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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
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US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
BR112020007790A2 (pt) * 2017-10-20 2020-10-20 Research Institute At Nationwide Children's Hospital métodos e materiais para terapia genética com nt-3
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US12491265B2 (en) 2018-06-18 2025-12-09 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
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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
JP2022521776A (ja) * 2019-02-26 2022-04-12 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 Β－サルコグリカンのアデノ随伴ウイルスベクター送達および筋ジストロフィーの治療
MY210230A (en) * 2019-08-21 2025-09-04 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
EP4117783A4 (en) * 2019-12-16 2024-08-21 Research Institute at Nationwide Children's Hospital COMPOSITIONS AND METHODS FOR RESTORING AND MAINTAINING DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX (DAPC)
CA3182313A1 (en) * 2020-06-19 2021-12-23 Isabelle Richard Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
CN119731321A (zh) 2022-06-24 2025-03-28 图恩疗法股份有限公司 通过靶向基因阻遏减少低密度脂蛋白的组合物、系统和方法
WO2025132132A1 (en) 2023-12-18 2025-06-26 F. Hoffmann-La Roche Ag Skeletal muscle-specific promoters
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