TY  - JOUR
AU  - Colella, Pasqualina
AU  - Sellier, Pauline
AU  - Gomez, Manuel J
AU  - Biferi, Maria G
AU  - Tanniou, Guillaume
AU  - Guerchet, Nicolas
AU  - Cohen-Tannoudji, Mathilde
AU  - Moya-Nilges, Maryse
AU  - van Wittenberghe, Laetitia
AU  - Daniele, Natalie
AU  - Gjata, Bernard
AU  - Krijnse-Locker, Jacomina
AU  - Collaud, Fanny
AU  - Simon-Sola, Marcelo
AU  - Charles, Severine
AU  - Cagin, Umut
AU  - Mingozzi, Federico
PY  - 2020
DO  - 10.1016/j.ebiom.2020.103052
UR  - http://hdl.handle.net/20.500.12105/11172
AB  - Pompe disease (PD) is a neuromuscular disorder caused by deficiency of acidalpha-glucosidase (GAA), leading to motor and respiratory dysfunctions. Available Gaa knock-out (KO) mouse models do not accurately mimic PD, particularly its highly impaired...
LA  - eng
PB  - Elsevier
TI  - Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.
TY  - journal article
ER  -