GENE THERAPY FOR TREATING CDKL5 DEFICIENCY DISORDER

GENE THERAPY FOR TREATING CDKL5 DEFICIENCY DISORDER

This present disclosure provides adeno-associated viral vectors, recombinant adeno-associated vims (rAAV), and methods of their use in gene therapy for treating CDKL5 deficiency disorder (CDD). Also provided are pharmaceutical compositions comprising an rAAV of the invention and a pharmaceutically a...

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Bibliographische Detailangaben
Hauptverfasser: Daugherty, Sean Christopher, Stoica, Lorelei Ioana, Fyffe-Maricich, Sharyl Lynne, Fuller, Matthew Scott, Wright, Margaret Caroline, Craig, Stewart
Format: Patent
Sprache:eng
Schlagworte:
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Zusammenfassung:This present disclosure provides adeno-associated viral vectors, recombinant adeno-associated vims (rAAV), and methods of their use in gene therapy for treating CDKL5 deficiency disorder (CDD). Also provided are pharmaceutical compositions comprising an rAAV of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the treatment of CDD caused by mutations in CDKL.