The Krainer team, in collaboration with a team at Isis Pharmaceuticals, surveyed a large number of antisense oligonucleotides (ASOs) and found that some of these ASOs are able to correct the mRNA splicing defect in cultured cells from SMA patients. (smafoundation.org)
March 13, 2007 — Cold Spring Harbor, New York — RNA splicing antisense technology studied at Cold Spring Harbor Laboratory (CSHL) effectively corrected an mRNA splicing defect found in spinal muscular atrophy (SMA) patients, and is now ready to be tested in mouse models. (smafoundation.org)
PRMT5 depletion led to a range of splicing defects linked to the retarded tumor growth. (sciencedaily.com)