Systems and methods for treating hemoglobinopathies

Genome editing systems, guide RNA and CRISPR-mediated methods are provided for altering portions of HBG1 and HBG2 loci in a cell and increasing expression of fetal hemoglobin. 提供了基因组编辑系统、向导RNA和CRISPR介导的方法，用于改变细胞中的HBG1和HBG2基因座的部分并且增加胎儿血红蛋白的表达。...

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Bibliographic Details
Main Author CHANG KWANHO
Format Patent
LanguageChinese
English
Published 26.04.2024
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
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Summary:Genome editing systems, guide RNA and CRISPR-mediated methods are provided for altering portions of HBG1 and HBG2 loci in a cell and increasing expression of fetal hemoglobin. 提供了基因组编辑系统、向导RNA和CRISPR介导的方法，用于改变细胞中的HBG1和HBG2基因座的部分并且增加胎儿血红蛋白的表达。
Bibliography:Application Number: CN202280058557