GENE THERAPY FOR TREATING CDKL5 DEFICIENCY DISORDER

This present disclosure provides adeno-associated viral vectors, recombinant adeno-associated virus (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...

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Bibliographische Detailangaben
Hauptverfasser:	FYFFE-MARICICH, SHARYL LYNNE, WRIGHT, MARGARET CAROLINE, DAUGHERTY, SEAN CHRISTOPHER, STOICA, LORELEI IOANA, CRAIG, STEWART, FULLER, MATTHEW SCOTT
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	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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Beschreibung
Zusammenfassung:	This present disclosure provides adeno-associated viral vectors, recombinant adeno-associated virus (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. La présente invention concerne des vecteurs viraux adéno-associés, un virus adéno-associé recombinant (rAAV), et des procédés d'utilisation de ceux-ci en thérapie génique pour le traitement d'un trouble du déficit en CDKL5 (CDD). L'invention concerne également des compositions pharmaceutiques comprenant un rAAV de l'invention et un support ou excipient pharmaceutiquement acceptable. Ces compositions pharmaceutiques peuvent être utiles en thérapie génique pour le traitement de CDD provoquée par des mutations dans CDKL.