HBB modulating compositions and methods

The present disclosure provides compositions, systems, and methods for targeting, editing, modifying, or manipulating a host cell genome at one or more locations of a DNA sequence in a cell, tissue, or subject, for example. Genetically modified systems for the treatment of sickle cell disease (SCD)...

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Hauptverfasser:	MCALLISTER GARY DALE, STEINBERG BARBARA ELAINE, FU YI, KOTLAR RYAN M, APPONI LUCIANO HENRIQUE, SANCHEZ, CL, RAY ANINDA, MENT, KIM KYUNGNAM, ROQUET NICOLAS, ALTSHULER, RICHARD, C, BOTHMER, ALAN, H, ABERNATTI DAVID GERARD, CHOI DANIEL R, CATALER-RAMUSINO CHRISTOPHER G S, HITRICK, ROBERT, J, SALOMON WALTER E, QUERBES WILLIAM, WANG ZHONGZE
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:	The present disclosure provides compositions, systems, and methods for targeting, editing, modifying, or manipulating a host cell genome at one or more locations of a DNA sequence in a cell, tissue, or subject, for example. Genetically modified systems for the treatment of sickle cell disease (SCD) are described. 本披露提供了例如用于在细胞、组织或受试者中的DNA序列的一个或多个位置处靶向、编辑、修饰或操纵宿主细胞基因组的组合物、系统和方法。描述了用于治疗镰状细胞病(SCD)的基因修饰系统。