Article

Duchenne muscular dystrophy treatment with lentiviral vector containing mini-dystrophin gene in vivo

2023-02-22

Abstract excerpt

Duchenne muscular dystrophy is an incurable X linked recessive genetic disease caused by mutations in the dystrophin gene. Many researchers aimed at restoring truncated dystrophin via viral vectors. But the low package capacity and short duration of vectors hampered their clinical application. For these reasons, we constructed four lentiviral vectors, which contained truncated and sequence-optimized dystrophin gen...

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Literature Corpus work: 2a3a32d9-9868-5742-abb1-cfe0dc8a2dc0
DOI: 10.22541/au.167706007.78963220/v1

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