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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Hauptverfasser:	FYFFE-MARICICH Sharyl Lynne, DAUGHERTY Sean Christopher, STOICA Lorelei Ioana, CRAIG Stewart, FULLER Matthew Scott, WRIGHT Margaret Caroline
Format:	Patent
Sprache:	eng ; spa
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. Esta presente descripción provee vectores virales adeno-asociados, virus recombinantes adeno-asociados (rAAV) y métodos para usarse en terapia génica para tratamiento de trastorno por deficiencia de CDKL5(CDD); también se proporcionan composiciones farmacéuticas que comprenden un rAAV de la invención y un portador o excipiente farmacéuticamente aceptable; estas composiciones farmacéuticas pueden ser útiles en terapia génica para el tratamiento de CDD causado por mutaciones en CDKL.