Mai K. ElMallah

Adeno‐associated virus–delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model

Lorelei Stoica, Sophia H. Todeasa, Gabriela Toro Cabrera et al. · Annals of Neurology · 2016 · 107 citations · Full text

Therapeutic rAAVrh10 Mediated <i>SOD1</i> Silencing in Adult <i>SOD1</i> ^G93A Mice and Nonhuman Primates

Florie Borel, Gwladys Gernoux, Brynn H. Cardozo et al. · Human Gene Therapy · 2015 · 103 citations · Full text

Editing out five <i>Serpina1</i> paralogs to create a mouse model of genetic emphysema

Florie Borel, Huaming Sun, Marina Zieger et al. · Proceedings of the National Academy of Sciences · 2018 · 88 citations · Full text

Sustained Correction of Motoneuron Histopathology Following Intramuscular Delivery of AAV in Pompe Mice

Mai K. ElMallah, Darin J. Falk, Sushrusha Nayak et al. · Molecular Therapy · 2013 · 84 citations · Full text

Peripheral nerve and neuromuscular junction pathology in Pompe disease

Darin J. Falk, Adrian G. Todd, Sooyeon Lee et al. · Human Molecular Genetics · 2014 · 83 citations · Full text