CO2018012082A2 - Administración de vectores del virus adenoasociado de β-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular - Google Patents

Administración de vectores del virus adenoasociado de β-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular

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Publication number
CO2018012082A2
CO2018012082A2 CONC2018/0012082A CO2018012082A CO2018012082A2 CO 2018012082 A2 CO2018012082 A2 CO 2018012082A2 CO 2018012082 A CO2018012082 A CO 2018012082A CO 2018012082 A2 CO2018012082 A2 CO 2018012082A2
Authority
CO
Colombia
Prior art keywords
sarcoglycan
muscular dystrophy
microarn
adeno
treatment
Prior art date
Application number
CONC2018/0012082A
Other languages
English (en)
Inventor
Louise Rodino-Klapac
Jerry R Mendell
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 CO2018012082A2 publication Critical patent/CO2018012082A2/es

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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • 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
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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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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    • 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
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    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Abstract

En la presente se describen vectores AAV recombinantes que comprenden una secuencia de polinucleótidos que comprende β-sarcoglicano 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 β-sarcoglicano y miR-29c a un sujeto mamífero que padece una distrofia muscular.
CONC2018/0012082A 2016-04-15 2018-11-08 Administración de vectores del virus adenoasociado de β-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular CO2018012082A2 (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 (1)

Publication Number Publication Date
CO2018012082A2 true CO2018012082A2 (es) 2019-02-08

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CONC2018/0012082A CO2018012082A2 (es) 2016-04-15 2018-11-08 Administración de vectores del virus adenoasociado de β-sarcoglicano y microarn-29 y el tratamiento de la distrofia muscular

Country Status (17)

Country Link
US (2) US11358993B2 (es)
EP (1) EP3442600B1 (es)
JP (2) JP7079733B2 (es)
KR (1) KR102502091B1 (es)
CN (2) CN117384964A (es)
AU (2) AU2017250756B2 (es)
BR (1) BR112018071167A2 (es)
CA (1) CA3021057A1 (es)
CO (1) CO2018012082A2 (es)
IL (1) IL262262B2 (es)
MA (1) MA44681A (es)
MX (2) MX2018012605A (es)
MY (1) MY195439A (es)
SA (1) SA518400245B1 (es)
SG (1) SG11201809029RA (es)
WO (1) WO2017180976A1 (es)
ZA (1) ZA201806863B (es)

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