Cerebellar Astrocyte Transduction as Gene Therapy for Megalencephalic Leukoencephalopathy

Megalencephalic leukoencephalopathy with subcortical cysts (MLC) is a rare genetic disorder belonging to the group of vacuolating leukodystrophies. It is characterized by megalencephaly, loss of motor functions, epilepsy, and mild mental decline. In brain biopsies of MLC patients, vacuoles were obse...

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Xehetasun bibliografikoak
Argitaratua izan da:Neurotherapeutics
Egile Nagusiak: Sánchez, Angela, García-Lareu, Belén, Puig, Meritxell, Prat, Esther, Ruberte, Jesús, Chillón, Miguel, Nunes, Virginia, Estévez, Raul, Bosch, Assumpció
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Springer International Publishing 2020
Gaiak:
Original Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC7851290/
https://ncbi.nlm.nih.gov/pubmed/32372403
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s13311-020-00865-y
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