Dose escalation pre-clinical trial of novel DOK7-AAV in mouse model of DOK7 congenital myasthenia

Authors
Cossins, Judith, Kozma, Imre, Canzonetta, Claudia, Hawkins, Al, Beeson, David, Sepulveda, Patricio, Dong, Yin Yao


Lab

Journal
Brain Communications

Abstract
Cossins et al. show that a novel adeno-associated viral gene therapy rescues a severe mouse model of DOK7 congenital myasthenia, making them as strong as w

Keywords/Topics
congenital myasthenia; gene therapy; DOK7-AAV; pre-clinical trial; neuromuscular junction

BIOSEB Instruments Used:
Grip strength test (BIO-GS4)

Source :

https://academic.oup.com/braincomms/article-abstract/7/1/fcaf046/7990910

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