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

• Main Authors: Stephanie Tantzer (Author), Karen Sperle (Author), Kaitlin Kenaley (Author), Jennifer Taube (Author), Grace M. Hobson (Author)
• Format: Knjiga
• Izdano: Elsevier, 2018-09-01T00:00:00Z.
• Teme: article
• Online dostop: Connect to this object online.

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)....