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Peptide-based therapeutic intervention for Cardiac Ryanopathies
TS-062097 — Novel polypeptides and polynucleotides targeting RyR2 to address dysregulated Ca2+ release.
The cardiac ryanodine receptor channel 2 (RyR2) is known as the Ca2+ release channel of the sarcoplasmic reticulum (SR), and is believed to be a good therapeutic target in a group of certain heart diseases, known as cardiac ryanopathies. A number of mutations in the RyR2 gene have been linked to a w…
  • College: College of Medicine (COM)
  • Inventors: Singh, Harpreet
  • Licensing Officer: Schultz, Teri

Antisense Oligonucleotide Development for Treatment of Spinal Muscular Atrophy
TS-037741 — The SMN2 gene contains sequences that regulate the level of inclusion of SMN exon7 in SMN mRNA, and some of these act in a negative manner. Antisense oligonucleotides to block the negative regulating protein binding to these sites can increase inclusion of SMN exon 7 and thus, the amount of SMN being produced.
Spinal muscular atrophy (SMA) is an inherited disease affecting the central nervous system, peripheral nervous system, and skeletal muscle. It is present primarily in infants and children but can also develop in adults. About 10,000 to 25,000 children and adults are living with the disease in the …
  • College: College of Medicine (COM)
  • Inventors: Burghes, Arthur; McGovern, Vicki; Prior, Thomas
  • Licensing Officer: He, Panqing

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