AUF1 GENE THERAPY FOR LIMB GIRDLE MUSCULAR DYSTROPHY

Provided are methods of treating or ameliorating the symptoms of limb girdle muscular dystrophy by administration of therapeutically effective doses of adeno-associated (AAV) or recombinant adeno-associated viruses (rAAV) containing a transgene encoding AU- rich element-binding factor 1 (AUF1) effec...

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Bibliographische Detailangaben
Hauptverfasser:	DANOS, Olivier, KARUMUTHIL-MELETHIL, Subha, QIAO, Chunping, FOLTZ, Steven, ABBADI, Dounia, ELLIOTT, Kirk, LIU, Ye, SCHNEIDER, Robert
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS OR TEST PAPERS THEREFOR COMPOSITIONS THEREOF CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL ORENZYMOLOGICAL PROCESSES CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEICACIDS OR MICROORGANISMS MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING ORGANIC CHEMISTRY PEPTIDES PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROCESSES OF PREPARING SUCH COMPOSITIONS PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Provided are methods of treating or ameliorating the symptoms of limb girdle muscular dystrophy by administration of therapeutically effective doses of adeno-associated (AAV) or recombinant adeno-associated viruses (rAAV) containing a transgene encoding AU- rich element-binding factor 1 (AUF1) effective to treat the limb girdle muscular dystrophy. Also provided are AAV and rAAV vectors encoding AUF1 proteins. L'invention concerne des méthodes de traitement ou d'amélioration des symptômes de la dystrophie musculaire des ceintures par administration de doses thérapeutiquement efficaces de virus adéno-associés (AAV) ou de virus adéno-associés recombinants (rAAV) contenant un transgène codant pour le facteur 1 de liaison à un élément riche en AU (AUF1) efficace pour traiter la dystrophie musculaire des ceintures. L'invention concerne également des vecteurs AAV et rAAV codant pour des protéines AUF1.