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

Provided herein are adeno-associated virus (AAV) compositions for correcting a mutation in a beta globin gene (HBB) gene and methods of using the same to correct an HBB gene mutation in a cell. Also provided are packaging systems for making the adeno-associated virus compositions. 本文提供了用于校正β珠蛋白(HBB)...

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Hauptverfasser: SMITH LAURA JANE, WONG KAMEHAMEHA K, WRIGHT JASON BOKE, DOLLIVE SERENA NICOLE, SEYMOUR ALBERT BARNES, CHATTERJEE SASWATI, ST MARTIN THIA BABOVAL, MCSWIGGEN JAMES ANTHONY, BENHAJSALAH MARWA, PROUT JAIME
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creator SMITH LAURA JANE
WONG KAMEHAMEHA K
WRIGHT JASON BOKE
DOLLIVE SERENA NICOLE
SEYMOUR ALBERT BARNES
CHATTERJEE SASWATI
ST MARTIN THIA BABOVAL
MCSWIGGEN JAMES ANTHONY
BENHAJSALAH MARWA
PROUT JAIME
description Provided herein are adeno-associated virus (AAV) compositions for correcting a mutation in a beta globin gene (HBB) gene and methods of using the same to correct an HBB gene mutation in a cell. Also provided are packaging systems for making the adeno-associated virus compositions. 本文提供了用于校正β珠蛋白(HBB)基因中的突变的腺相关病毒(AAV)组合物和使用所述组合物校正细胞中的HBB基因突变的方法。还提供了用于制备所述腺相关病毒组合物的包装系统。
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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
SPIRITS
VINEGAR
WINE
title ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING HBB GENE FUNCTION AND METHODS OF USE THEREOF
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