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MX2018012605A - Administracion de vectores del virus adenoasociado de b-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular. - Google Patents

Administracion de vectores del virus adenoasociado de b-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular.

Info

Publication number
MX2018012605A
MX2018012605A MX2018012605A MX2018012605A MX2018012605A MX 2018012605 A MX2018012605 A MX 2018012605A MX 2018012605 A MX2018012605 A MX 2018012605A MX 2018012605 A MX2018012605 A MX 2018012605A MX 2018012605 A MX2018012605 A MX 2018012605A
Authority
MX
Mexico
Prior art keywords
muscular dystrophy
vectors
sarcoglicano
microarn
adeno
Prior art date
Application number
MX2018012605A
Other languages
English (en)
Other versions
MX391208B (es
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 MX2018012605A publication Critical patent/MX2018012605A/es
Publication of MX391208B publication Critical patent/MX391208B/es

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • A61K48/0008—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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
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Abstract

En la presente se describen vectores AAV recombinantes que comprenden una secuencia de polinucleótidos que comprende ß-sarcogIicano y métodos para usar los vectores recombinantes para reducir o prevenir la fibrosis en un sujeto mamífero que padece una distrofia muscular. También se describen en la presente tratamientos conjuntos que comprenden administrar vectores VAA que expresan ß-sarcogIicano y miR-29c a un sujeto mamífero que padece una distrofia muscular.
MX2018012605A 2016-04-15 2017-04-14 Vectores del virus adenoasociado de b-sarcoglicano y microarn-29 y el uso de los mismos en la distrofia muscular. MX391208B (es)

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

Publications (2)

Publication Number Publication Date
MX2018012605A true MX2018012605A (es) 2019-07-04
MX391208B MX391208B (es) 2025-03-21

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018012605A MX391208B (es) 2016-04-15 2017-04-14 Vectores del virus adenoasociado de b-sarcoglicano y microarn-29 y el uso de los mismos en la distrofia muscular.
MX2022003857A MX2022003857A (es) 2016-04-15 2018-10-15 Vectores del virus adenoasociado de b-sarcoglicano y microarn-29 y el uso de los mismos en la distrofia muscular.

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MX2022003857A MX2022003857A (es) 2016-04-15 2018-10-15 Vectores del virus adenoasociado de b-sarcoglicano y microarn-29 y el uso de los mismos en la distrofia muscular.

Country Status (28)

Country Link
US (3) US11358993B2 (es)
EP (2) EP4407035A3 (es)
JP (2) JP7079733B2 (es)
KR (1) KR102502091B1 (es)
CN (2) CN109121395B (es)
AU (2) AU2017250756B2 (es)
BR (1) BR112018071167A2 (es)
CA (1) CA3021057A1 (es)
CO (1) CO2018012082A2 (es)
DK (1) DK3442600T5 (es)
ES (1) ES2979174T3 (es)
FI (1) FI3442600T3 (es)
HR (1) HRP20240580T1 (es)
HU (1) HUE066442T2 (es)
IL (1) IL262262B2 (es)
LT (1) LT3442600T (es)
MA (1) MA44681A (es)
MX (2) MX391208B (es)
MY (1) MY195439A (es)
PL (1) PL3442600T3 (es)
PT (1) PT3442600T (es)
RS (1) RS65490B1 (es)
SA (1) SA518400245B1 (es)
SG (1) SG11201809029RA (es)
SI (1) SI3442600T1 (es)
SM (1) SMT202400169T1 (es)
WO (1) WO2017180976A1 (es)
ZA (1) ZA201806863B (es)

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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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EP3596112A2 (en) 2017-03-17 2020-01-22 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
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EP4168052A2 (en) * 2020-06-19 2023-04-26 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
JP2023059858A (ja) 2021-10-15 2023-04-27 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 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
JP2025524469A (ja) 2022-06-24 2025-07-30 チューン セラピューティクス インコーポレイテッド 標的を定めた遺伝子抑制を介して低密度リポタンパク質を低減するための組成物、系、及び方法
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