Columbia Technology Ventures

RNA regulation of the SLC2A1 gene for treatment of Glut1 deficiency

This technology is a naturally occurring non-coding RNA that regulates the SLC2A1 gene, allowing for therapeutic regulation of Glut1 for treatment of diseases in which Glut1 is under-expressed.

Unmet Need: Treatment for Glut1 deficiency syndrome and related disorders

Mutation of the Glut1 protein can lead to impaired transport of glucose across the blood-brain barrier. Despite the implications of Glut1 dysfunction in a variety of diseases ranging from Glut1 deficiency syndrome (Glut1 DS) to Alzheimer’s disease, retinitis pigmentosa, cancer, and diabetes, there are currently no therapeutic treatments for aberrations in Glut1 protein levels. Currently, management of Glut1DS involves the adoption of a ketogenic diet and regular use of anticonvulsant drugs. While Glut1 DS is a relatively rare disorder, regulation of Glut1 expression also has significant implications for neurological and metabolic disease patients.

The Technology: RNA therapy for treatment of Glut1 abnormalities

This technology is a naturally occurring long non-coding RNA that is an antisense transcript to the SLC2A1 (Glut1) gene. Found in two isoforms, these RNAs have demonstrated the ability to regulate expression of Glut1, by either decreasing or increasing Glut1 depending on whether the gene is silenced or overexpressed. This RNA target offers the potential for a powerful treatment for diseases caused by Glut1 abnormalities.

This technology has been validated in fibroblast lines and primary brain endothelial cells.

Applications:

  • Treatment of Glut1 DS and metabolic diseases
  • Research tool for analysis of mechanisms relating to glucose transport
  • RNA-based biomarker of Glut1-overexpressing brain tumor cells
  • Potential treatment of other diseases exhibiting abnormal levels of Glut1 protein such as cancer, diabetes, retinitis pigmentosa, cerebral ischemia, and Alzheimer’s disease
  • Enhancement of blood-brain barrier permeation via the leveraging of Glut1 protein

Advantages:

  • Specific target to control disease without lifestyle changes
  • Compatible with RNAi therapeutic platforms
  • Provides potential treatment for a wide variety of metabolic diseases

Lead Inventor:

Umrao Monani, Ph.D.

Patent Information:

Patent Status

Related Publications:

Tech Ventures Reference:

  • IR CU17177

  • Licensing Contact: Ron Katz