SYSTEMS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES

Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci in cells and increasing expression of fetal hemoglobin. Systèmes d'édition génomique, ARN guides et procédés médiés par CRISPR pour modifier des parties des loci HBG1 et...

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1. Verfasser:	CHANG, KAIHSIN
Format:	Patent
Sprache:	eng ; fre
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:	Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci in cells and increasing expression of fetal hemoglobin. Systèmes d'édition génomique, ARN guides et procédés médiés par CRISPR pour modifier des parties des loci HBG1 et HBG2 dans les cellules et augmenter l'expression de l'hémoglobine ftale.