Results 331 to 340 of about 9,766,746 (345)

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

Nature Medicine, 2019
Christopher E Nelson, Yaoying Wu, Matthew A Waller   +2 more
exaly  

Induced pluripotent stem cells from a spinal muscular atrophy patient

Nature, 2008
Allison D Ebert, Christian L Lorson, Clive N Svendsen   +2 more
exaly  

Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

Nature Neuroscience, 2017
D. Corey
semanticscholar   +1 more source

Therapeutic developments for Duchenne muscular dystrophy

Nature Reviews Neurology, 2019
Ingrid E C Verhaart, Annemieke Aartsma-Rus   +1 more
exaly  

Defective membrane repair in dysferlin-deficient muscular dystrophy

Nature, 2003
Steven S Vogel, Chien-Chang Chen, Kevin P Campbell   +2 more
exaly  

Genetic and pharmacologic inhibition of mitochondrial-dependent necrosis attenuates muscular dystrophy

Nature Medicine, 2008
Douglas P Millay, Christopher P Baines, Elisabeth R Barton   +2 more
exaly  

Spinal muscular atrophy — insights and challenges in the treatment era

Nature Reviews Neurology, 2020
Eugenio Mercuri, Maria Carmela Pera, Richard S Finkel   +2 more
exaly  

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

Nature Medicine, 2020
exaly  

Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy

Nature Medicine, 2002
Scott Q Harper, Dongsheng Duan, John F Engelhardt   +2 more
exaly  

Advances in therapy for spinal muscular atrophy: promises and challenges

Nature Reviews Neurology, 2018
Ewout J N Groen, Kevin Talbot, Thomas Gillingwater   +2 more
exaly