Future Prospects of Gene Therapy for Friedreich’s Ataxia

Friedreich’s ataxia is an autosomal recessive neurogenetic disease that is mainly associated with atrophy of the spinal cord and progressive neurodegeneration in the cerebellum. The disease is caused by a GAA-expansion in the first intron of the frataxin gene leading to a decreased level of frataxin...

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Detalles Bibliográficos
Autores principales: Ocana-Santero, Gabriel, Díaz-Nido, Javier, Herranz-Martín, Saúl
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7918362/
https://www.ncbi.nlm.nih.gov/pubmed/33670433
http://dx.doi.org/10.3390/ijms22041815

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7918362/
https://www.ncbi.nlm.nih.gov/pubmed/33670433
http://dx.doi.org/10.3390/ijms22041815

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