COMPOSITIONS AND METHODS FOR EXPRESSING FACTOR IX FOR HEMOPHILIA B THERAPY

Nucleic acid constructs and compositions that allow insertion of a FIX coding sequence into a target genomic locus such as an endogenous ALB locus and/or expression of the FIX coding sequence are provided. The nucleic acid constructs and compositions can be used in methods of introducing a F9 nuclei...

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Main Authors SAMAI, Poulami, CALAFATI, Philip, CYGNAR, Katherine, KYRATSOUS, Christos, SABIN, Leah, PEFANIS, Evangelos, BAIK, Andrew, MOLLER-TANK, Sven, KATAKOWSKI, Joseph
Format Patent
LanguageEnglish
French
German
Published 04.09.2024
Subjects
CHEMISTRY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PEPTIDES
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Summary:Nucleic acid constructs and compositions that allow insertion of a FIX coding sequence into a target genomic locus such as an endogenous ALB locus and/or expression of the FIX coding sequence are provided. The nucleic acid constructs and compositions can be used in methods of introducing a F9 nucleic acid into a cell, methods of integration of a F9 nucleic acid into a target genomic locus, methods of expression of FIX in a cell, and in methods of treating hemophilia B or FIX deficiency in a subject.
Bibliography:Application Number: EP20220818964