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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