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

• मुख्य लेखकों: Stephanie Tantzer (लेखक), Karen Sperle (लेखक), Kaitlin Kenaley (लेखक), Jennifer Taube (लेखक), Grace M. Hobson (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Elsevier, 2018-09-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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)....