Article
Antisense oligonucleotides targeting exon 11 are able to partially rescue the Neurofibromatosis Type 2 phenotype <i>in vitro</i>
2022-02-11
Abstract excerpt
Neurofibromatosis type 2 (NF2) is an autosomal dominant condition caused by loss of function variants in the NF2 gene, which codes for the protein Merlin, and characterized by the development of multiple tumours of the nervous system. The clinical presentation of the disease is variable and related to the type of the inherited germline variant. Here, we tested if PMOs could be used to correct the splice signallin...
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Identifiers and source
- Literature Corpus work
- e2f5ba99-9931-5069-b45a-74899eb74ec4
- DOI
- 10.1101/2022.02.11.479859
