AR122165A1 - ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING PAH GENE FUNCTION AND METHODS OF USING THESE - Google Patents

ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING PAH GENE FUNCTION AND METHODS OF USING THESE

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Publication number
AR122165A1
AR122165A1 ARP210101411A ARP210101411A AR122165A1 AR 122165 A1 AR122165 A1 AR 122165A1 AR P210101411 A ARP210101411 A AR P210101411A AR P210101411 A ARP210101411 A AR P210101411A AR 122165 A1 AR122165 A1 AR 122165A1
Authority
AR
Argentina
Prior art keywords
methods
adeno
gene function
associated virus
virus compositions
Prior art date
Application number
ARP210101411A
Other languages
Spanish (es)
Inventor
Jason Boke Wright
Danielle Lauren Sookiasian
Martin Thia Baboval St
Omar Francone
Albert Barnes Seymour
Original Assignee
Homology Medicines Inc
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 Homology Medicines Inc filed Critical Homology Medicines Inc
Publication of AR122165A1 publication Critical patent/AR122165A1/en

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

En la presente se proporcionan composiciones de virus adenoasociados recombinantes (rAAV) que pueden restaurar la función del gen de la fenilalanina hidroxilasa (PAH) en las células, y métodos para el uso de estas para tratar enfermedades asociadas con la reducción de la función del gen PAH (por ejemplo, PKU). También se proporcionan ácidos nucleicos, vectores, sistemas de empaquetamiento, y métodos para fabricar las composiciones de virus adenoasociados.Provided herein are recombinant adeno-associated virus (rAAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cells, and methods of using these to treat diseases associated with reduced gene function. PAH (for example, PKU). Nucleic acids, vectors, packaging systems, and methods for making the adeno-associated virus compositions are also provided.

ARP210101411A 2020-05-27 2021-05-26 ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING PAH GENE FUNCTION AND METHODS OF USING THESE AR122165A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063030341P 2020-05-27 2020-05-27

Publications (1)

Publication Number Publication Date
AR122165A1 true AR122165A1 (en) 2022-08-24

Family

ID=83218668

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210101411A AR122165A1 (en) 2020-05-27 2021-05-26 ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING PAH GENE FUNCTION AND METHODS OF USING THESE

Country Status (1)

Country Link
AR (1) AR122165A1 (en)

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