Available Technologies
# of Displayed Technologies: 3 / 3
Categories
Utilizing Antisense Oligonucleotides to Modulate MDM2 Alternative Splicing
TS-000194 —
Investigators at Nationwide Children's Hospital have developed a novel splicing corrective treatment to modulate the splicing of p53-modifier Murine Double Minute 2 (MDM2) in cancer.
Murine Double Minute 2 (MDM2) is an E3 ubiquitin ligase and negative regulator of the tumor suppressor protein p53. Under normal conditions, MDM2 is constitutively spliced to generate a full-length protein and promotes the proteasome-mediated degradation of p53. However, under stress MDM2 undergoes …
- College:
- Inventors: Chandler, Dawn; Comiskey, Daniel
- Licensing Officer: Murrah, Kyle
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
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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
