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Antisense oligonucleotides targeting exon 11 are able to partially rescue the NF2-related schwannomatosis phenotype in vitro

NF2-related schwannomatosis (NF2-related SWN) is an autosomal dominant condition caused by loss of function variants in the NF2 gene, which codes for the protein Merlin and is characterized by the development of multiple tumors of the nervous system. The clinical presentation of the disease is varia...

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Detalles Bibliográficos
Autores principales: Catasús, Núria, Rosas, Inma, Bonache, Sandra, Negro, Alex, Torres-Martin, Miguel, Plana-Pla, Adrià, Salvador, Hector, Serra, Eduard, Blanco, Ignacio, Castellanos, Elisabeth
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9678674/
https://www.ncbi.nlm.nih.gov/pubmed/36420221
http://dx.doi.org/10.1016/j.omtn.2022.10.026