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
Format:	Patent
Sprache:	chi ; eng
Schlagworte:	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
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Beschreibung
Zusammenfassung:	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。