A METHOD FOR IN VIVO GENE THERAPY TO CURE SCD WITHOUT MYELOABLATIVE TOXICITY

Disclosures herein are directed to compositions and methods for increasing gamma globinexpression in a cell or subject by suppressing expression of Klf1. Also described are methods of treating or ameliorating-hemoglobinopathies. The compositions are administered to a subject as a therapeutic amount...

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Bibliographic Details
Main Authors BIEKER, James J, GLASSBERG, Jeffrey A
Format Patent
LanguageEnglish
French
German
Published 13.08.2025
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
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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Summary:Disclosures herein are directed to compositions and methods for increasing gamma globinexpression in a cell or subject by suppressing expression of Klf1. Also described are methods of treating or ameliorating-hemoglobinopathies. The compositions are administered to a subject as a therapeutic amount of a gene editing composition, including an RNA-guided endonuclease and one or more gRNA or sgRNA, or one or more transcription activator-like effector nucleases (TALENs), that targets an intron of a Klf1 gene locus in at least one cell in the subject to effect a deletion in intron, thereby reducing expression of Klf1.
Bibliography:Application Number: EP20220846850