COMPOSITIONS FOR AND METHODS OF GENE EDITING

Compositions and methods for treating a blood disorder in a subject comprising delivering a nucleic acid molecule including a nucleotide sequence encoding two to six guide RNAs (gRNAs) into a hematopoietic stem cell (HSC), a hematopoietic progenitor cell (HPC), or a population of hematopoietic stem...

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Bibliographische Detailangaben
Hauptverfasser:	FLOREA, Michael, WAGERS, Amy, KARIMZADEH, Alborz
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 ORGANIC CHEMISTRY PEPTIDES 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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Beschreibung
Zusammenfassung:	Compositions and methods for treating a blood disorder in a subject comprising delivering a nucleic acid molecule including a nucleotide sequence encoding two to six guide RNAs (gRNAs) into a hematopoietic stem cell (HSC), a hematopoietic progenitor cell (HPC), or a population of hematopoietic stem and progenitor cells (HSPCs) are described. L'invention concerne des compositions et des méthodes de traitement d'un trouble sanguin chez un sujet, comprenant l'administration d'une molécule d'acide nucléique comprenant une séquence nucléotidique codant pour deux à six ARN guides (ARNg) dans une cellule souche hématopoïétique (CSH), une cellule progénitrice hématopoïétique (CPH), ou une population de cellules souches et progénitrices hématopoïétiques (CSPH).