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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| creator | 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 |
| description | 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. |
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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.</abstract><oa>free_for_read</oa></addata></record> |
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| subjects | 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 |
| title | METHODS OF TREATING DUCHENNE MUSCULAR DYSTROPHY USING AAV MINI-DYSTROPHIN GENE THERAPY |
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