AR121770A1 - COMPOSITIONS OF ADENO-ASSOCIATED VIRUSES FOR THE TRANSFER OF THE IDS GENE AND THEIR METHODS OF USE - Google Patents

COMPOSITIONS OF ADENO-ASSOCIATED VIRUSES FOR THE TRANSFER OF THE IDS GENE AND THEIR METHODS OF USE

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Publication number
AR121770A1
AR121770A1 ARP210100900A ARP210100900A AR121770A1 AR 121770 A1 AR121770 A1 AR 121770A1 AR P210100900 A ARP210100900 A AR P210100900A AR P210100900 A ARP210100900 A AR P210100900A AR 121770 A1 AR121770 A1 AR 121770A1
Authority
AR
Argentina
Prior art keywords
compositions
methods
adeno
transfer
ids gene
Prior art date
Application number
ARP210100900A
Other languages
Spanish (es)
Inventor
Jacinthe Gingras
Kruti Patel
Laura Jane Smith
Yvonne White
Serena Nicole Dollive
Lieshout Laura Van
Brenda Burnham
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 AR121770A1 publication Critical patent/AR121770A1/en

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

Abstract

Se proporcionan composiciones de virus adenoasociados (AAV) que pueden restaurar la función del gen IDS en las células, y métodos para usar estas composiciones de AAV para tratar trastornos asociados con la reducción de la función del gen IDS (por ejemplo, síndrome de Hunter). También se proporcionan composiciones, sistemas y métodos para preparar las composiciones de AAV.Adeno-associated virus (AAV) compositions that can restore IDS gene function in cells, and methods of using these AAV compositions to treat disorders associated with reduced IDS gene function (eg, Hunter syndrome) are provided. . Compositions, systems, and methods for preparing the AAV compositions are also provided.

ARP210100900A 2020-04-06 2021-04-06 COMPOSITIONS OF ADENO-ASSOCIATED VIRUSES FOR THE TRANSFER OF THE IDS GENE AND THEIR METHODS OF USE AR121770A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063005833P 2020-04-06 2020-04-06

Publications (1)

Publication Number Publication Date
AR121770A1 true AR121770A1 (en) 2022-07-06

Family

ID=83218572

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210100900A AR121770A1 (en) 2020-04-06 2021-04-06 COMPOSITIONS OF ADENO-ASSOCIATED VIRUSES FOR THE TRANSFER OF THE IDS GENE AND THEIR METHODS OF USE

Country Status (1)

Country Link
AR (1) AR121770A1 (en)

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