ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING PAH GENE FUNCTION AND METHODS OF USE THEREOF

Provided herein are adeno-associated virus (AAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cell. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions. 本文提供了可以在细胞中恢复苯丙氨酸羟化酶(PAH)基因功能的腺相关病毒(AAV)组合物。还提供了使用所述A...

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Bibliographic Details
Main Authors AHMED SEEMIN S, PROUT JAIME M, DOLLIVE SERENA N, SOOKIASIAN DANIELLE L, WRIGHT JASON B, SEYMOUR ALBERT B, MCSWIGGEN JAMES A
Format Patent
LanguageChinese
English
Published 06.11.2020
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
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Summary:Provided herein are adeno-associated virus (AAV) compositions that can restore phenylalanine hydroxylase (PAH) gene function in cell. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions. 本文提供了可以在细胞中恢复苯丙氨酸羟化酶(PAH)基因功能的腺相关病毒(AAV)组合物。还提供了使用所述AAV组合物的方法以及用于制备所述AAV组合物的包装系统。
Bibliography:Application Number: CN201980021206