EA202190155A1 -
PRODUCTS BASED ON RECOMBINANT ADENO-ASSOCIATED VIRUSES AND METHODS FOR TREATMENT OF PELVIC-SHOULDER MUSCULAR DYSTROPHY TYPE 2A
- Google Patents
PRODUCTS BASED ON RECOMBINANT ADENO-ASSOCIATED VIRUSES AND METHODS FOR TREATMENT OF PELVIC-SHOULDER MUSCULAR DYSTROPHY TYPE 2A
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Application filed by Рисерч Инститьют Эт Нэшнуайд Чилдрен'С ХоспиталfiledCriticalРисерч Инститьют Эт Нэшнуайд Чилдрен'С Хоспитал
Priority claimed from PCT/US2019/039893external-prioritypatent/WO2020006458A1/en
Publication of EA202190155A1publicationCriticalpatent/EA202190155A1/en
Medicines That Contain Protein Lipid Enzymes And Other Medicines
(AREA)
Abstract
Изобретение относится к продуктам и способам для лечения тазово-плечевой мышечной дистрофии типа 2А. В этих способах рекомбинантные аденоассоциированные вирусы доставляют ДНК, кодирующую белок, имеющий активность кальпаина-3.The invention relates to products and methods for the treatment of type 2A pelvic-brachial muscular dystrophy. In these methods, recombinant adeno-associated viruses deliver DNA encoding a protein having calpain-3 activity.
EA202190155A2019-06-212019-06-28
PRODUCTS BASED ON RECOMBINANT ADENO-ASSOCIATED VIRUSES AND METHODS FOR TREATMENT OF PELVIC-SHOULDER MUSCULAR DYSTROPHY TYPE 2A
EA202190155A1
(en)
AAV VECTORS WITH PROMOTER MYELIN PROTEIN ZERO AND THEIR USE FOR THE TREATMENT OF DISEASES ASSOCIATED WITH SCHWANN CELLS SUCH AS CHARCOT-MARIETTE DISEASE