Allele-specific silencing as therapy for familial amyotrophic lateral sclerosis caused by the p.G376D TARDBP mutation

Amyotrophic lateral sclerosis is a neurodegenerative disease characterized by the degeneration of motor neurons. There is no treatment for this disease that affects the ability to move, eat, speak and finally breathe, causing death. In an Italian family, a heterozygous pathogenic missense variant ha...

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Detalles Bibliográficos
Autores principales: Romano, Roberta, De Luca, Maria, Del Fiore, Victoria Stefania, Pecoraro, Martina, Lattante, Serena, Sabatelli, Mario, La Bella, Vincenzo, Bucci, Cecilia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9897181/
https://www.ncbi.nlm.nih.gov/pubmed/36751500
http://dx.doi.org/10.1093/braincomms/fcac315

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9897181/
https://www.ncbi.nlm.nih.gov/pubmed/36751500
http://dx.doi.org/10.1093/braincomms/fcac315

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