AR118192A1 - ADMINISTRATION OF b-SARCOGLICAN WITH A VECTOR OF ADENO-ASSOCIATED VIRUS AND TREATMENT OF MUSCULAR DYSTROPHY - Google Patents

ADMINISTRATION OF b-SARCOGLICAN WITH A VECTOR OF ADENO-ASSOCIATED VIRUS AND TREATMENT OF MUSCULAR DYSTROPHY

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Publication number
AR118192A1
AR118192A1 ARP200100520A ARP200100520A AR118192A1 AR 118192 A1 AR118192 A1 AR 118192A1 AR P200100520 A ARP200100520 A AR P200100520A AR P200100520 A ARP200100520 A AR P200100520A AR 118192 A1 AR118192 A1 AR 118192A1
Authority
AR
Argentina
Prior art keywords
muscular dystrophy
vector
sarcoglican
adeno
administration
Prior art date
Application number
ARP200100520A
Other languages
Spanish (es)
Inventor
Jerry R Mendell
Louise Rodino-Klapac
Original Assignee
Res Institute At Nationwide Children’S 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 Institute At Nationwide Children’S Hospital filed Critical Res Institute At Nationwide Children’S Hospital
Publication of AR118192A1 publication Critical patent/AR118192A1/en

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Abstract

Se describen en el presente documento métodos para tratar la distrofia muscular que comprenden administrar un vector recombinante AAV (VAAr) scAAVrh74.MHCK7.hSGCB, métodos para expresar el gen del b-sarcoglicano en un paciente, composiciones farmacéuticas que comprenden el VAAr, y métodos para generar el VAAr.Described herein are methods for treating muscular dystrophy that comprise administering a recombinant AAV vector (AAVr) scAAVrh74.MHCK7.hSGCB, methods for expressing the b-sarcoglycan gene in a patient, pharmaceutical compositions comprising rAVa, and methods to generate the VAAr.

ARP200100520A 2019-02-26 2020-02-26 ADMINISTRATION OF b-SARCOGLICAN WITH A VECTOR OF ADENO-ASSOCIATED VIRUS AND TREATMENT OF MUSCULAR DYSTROPHY AR118192A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962810917P 2019-02-26 2019-02-26

Publications (1)

Publication Number Publication Date
AR118192A1 true AR118192A1 (en) 2021-09-22

Family

ID=78076096

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200100520A AR118192A1 (en) 2019-02-26 2020-02-26 ADMINISTRATION OF b-SARCOGLICAN WITH A VECTOR OF ADENO-ASSOCIATED VIRUS AND TREATMENT OF MUSCULAR DYSTROPHY

Country Status (1)

Country Link
AR (1) AR118192A1 (en)

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