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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Publicado en:Neurotherapeutics
Autores principales: Sánchez, Angela, García-Lareu, Belén, Puig, Meritxell, Prat, Esther, Ruberte, Jesús, Chillón, Miguel, Nunes, Virginia, Estévez, Raul, Bosch, Assumpció
Formato: Artigo
Lenguaje:Inglês
Publicado: Springer International Publishing 2020
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Original Article
Acceso en línea: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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