METHODS AND AAV VECTORS FOR IN VIVO TRANSDUCTION

The present disclosure relates generally to methods and adeno-associated virus (AAV) vectors for in vivo transduction. The disclosure is directed to methods for producing modified AAV capsid polypeptides and AAV vectors that are suitable for transduction of cells in vivo, and methods for screening A...

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Bibliographic Details
Main Authors LISOWSKI, Leszek, CABANES CREUS, Marti, HALLWIRTH, Claus Volker, GINN, Samantha Lee, ALEXANDER, Ian Edward
Format Patent
LanguageEnglish
French
German
Published 21.02.2024
Subjects
CHEMISTRY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PEPTIDES
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Summary:The present disclosure relates generally to methods and adeno-associated virus (AAV) vectors for in vivo transduction. The disclosure is directed to methods for producing modified AAV capsid polypeptides and AAV vectors that are suitable for transduction of cells in vivo, and methods for screening AAV vectors. The disclosure also relates to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules, AAV vectors comprising the capsid polypeptides, and nucleic acid vectors comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.
Bibliography:Application Number: EP20200834114