# of Displayed Technologies: 2 / 2

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Therapeutic Heat Shock Dulation of SMN levels in Spinal Muscular Atrophy (SMA)
TS-000193 — SMA currently has no treatment and is the result of an mRNA splicing defect which we have found can be corrected with heat shock. We propose using increased temperature, small molecule activators of heat shock response, or other mechanisms for inducing the heat shock response for treatment of SMA.
Spinal Muscular Atrophy (SMA) is a neurodegenerative disease that occurs in 1 of every 6,000 births, and is caused by low levels of the SMN protein. SMA patients have inherited deletions or mutations of SMN1, one of two genes encoding SMN. SMN2 contains a translationally silent single nucleotide swi…
  • College:
  • Inventors: Chandler, Dawn; Dominguez, Catherine
  • Licensing Officer: Murrah, Kyle

Transfected Cell Line for Drug Discovery Aimed at Splicing Correction
TS-000113 — Technology Overview Researchers at the Research Institute have developed a stably transfected cell line that expresses wild type survival motor neuron gene-2 (SMN2) that can be used as a drug discovery tool aimed at gene splicing correction. SMN2 is a potential therapeutic target for Proximal Spinal…
  • College:
  • Inventors: Chandler, Dawn; Bebee, Thomas; Gladman, Jordan
  • Licensing Officer: Murrah, Kyle

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