METHODS OF TREATING DUCHENNE MUSCULAR DYSTROPHY USING AAV MINI-DYSTROPHIN GENE THERAPY

The disclosure describes methods of treating humans with Duchenne muscular dystrophy by providing doses of an AAV9 vector that expresses a mini-dystrophin protein in transduced muscle cells. L'invention concerne des procédés de traitement d'êtres humains atteints d'une dystrophie musc...

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Hauptverfasser:	BINKS, Michael, MCDONNELL MOOREHEAD, Tara, WHITELEY, Laurence Oliver, YONG, Florence Hiu-Ling, SHAPKINA, Tatiana G, BEIDLER, David Roger, SANKAR, Savita, SHERLOCK, Sarah Paige, HUANG, Rong, RUNNELS, Herbert, BALDUS, Phoebe Arnold, NEUBERT, Hendrik, NEELAKANTAN, Srividya, DEMARCO, Suzanne C
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS OR TEST PAPERS THEREFOR CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL ORENZYMOLOGICAL PROCESSES ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEICACIDS OR MICROORGANISMS MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MUTATION OR GENETIC ENGINEERING ORGANIC CHEMISTRY PEPTIDES PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROCESSES OF PREPARING SUCH COMPOSITIONS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	The disclosure describes methods of treating humans with Duchenne muscular dystrophy by providing doses of an AAV9 vector that expresses a mini-dystrophin protein in transduced muscle cells. L'invention concerne des procédés de traitement d'êtres humains atteints d'une dystrophie musculaire de Duchenne par l'administration de doses d'un vecteur AAV9 qui exprime une protéine de mini-dystrophine dans des cellules musculaires transduites.