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

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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Bibliographic Details
Main Authors: Núria Catasús, Inma Rosas, Sandra Bonache, Alex Negro, Miguel Torres-Martin, Adrià Plana-Pla, Hector Salvador, Eduard Serra, Ignacio Blanco, Elisabeth Castellanos
Format: Article
Language:English
Published: Elsevier 2022-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: oligonucleotides: therapies and applications
neurofibromatosis type 2
antisense therapy
antisense oligomers
genotype-phenotype correlations
Merlin
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122002931

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http://www.sciencedirect.com/science/article/pii/S2162253122002931

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