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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Hauptverfasser: FLOREA, Michael, WAGERS, Amy, KARIMZADEH, Alborz
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creator FLOREA, Michael
WAGERS, Amy
KARIMZADEH, Alborz
description 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).
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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 COMPOSITIONS FOR AND METHODS OF GENE EDITING
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