Morpholino Antisense Oligomers as a Potential Therapeutic Option for the Correction of Alternative Splicing in PMD, SPG2, and HEMS

DNA variants of the proteolipid protein 1 gene (PLP1) that shift PLP1/DM20 alternative splicing away from the PLP1 form toward DM20 cause the allelic X-linked leukodystrophies Pelizaeus-Merzbacher disease (PMD), spastic paraplegia 2 (SPG2), and hypomyelination of early myelinating structures (HEMS)....

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Publicado en:Mol Ther Nucleic Acids
Main Authors: Tantzer, Stephanie, Sperle, Karen, Kenaley, Kaitlin, Taube, Jennifer, Hobson, Grace M.
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Gene & Cell Therapy 2018
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6036941/
https://ncbi.nlm.nih.gov/pubmed/30195779
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.05.019
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