Novel Channelrhodopsins for Optogenetics Gene Therapy

Description:

 Novel Channelrhodopsins for Optogenetics Gene Therapy
 

When expressed in targeted neurons, light-sensitive channelrhodopsin proteins (ChRs) have the ability to selectively manipulate neuronal activity in a light-dependent manner. Gene therapy using anion ChRs (ACRs) has the potential for treating excessive neuron firing in conditions such as Parkinson’s disease, neuropathic pain and epilepsy, while cation ChRs (CCRs) hold promise for vision restoration. Dr. John Spudich and his team at UTHealth have developed suites of ACRs and CCRs, which are orders of magnitude more efficient than currently available optogenetic tools and have the ability to effectively inhibit neuron firing.

Technology Overview

  • Anion channelrhodopsins (ACRs) that
    • Provide neuronal silencing
    • Hyperpolarize cultured animal cell membranes at < 1/1000 of the light intensity required by the most efficient currently available optogenetic protein
  • Cation ChRs (CCRs) containing mutations of the transmembrane helix 4 with
    • Superior photocurrent amplitudes
    • Enhanced Na+ selectivity
    • Enhanced Ca2+ conductance

Stage of Development
Humanized ACRs have been successfully expressed in mammalian neurons, and have further been validated by a number of laboratories working in various animal systems, including the targeted silencing of neurons in the living mouse brain.  

Clinical Applications
Our ChRs have the potential to be used as gene therapy-based therapeutics for diseases caused by aberrant neuronal activity or other cellular excitation, such as neurodegenerative disease, neuropathic pain, ocular disorders, epilepsy, and cardiac disorders. 
 

Intellectual Property Status
Issued and pending patents (US and foreign) on suites of anion and cation conducting channel-rhodopsins with varying spectral sensitivities
are available for licensing: 

  • US 10,519,205
  • US 9,676,836
  • CN 2021041200040170

Selected Publications

  • Science. 2015 Aug7;349(6248):647-50. PMID: 26113638; https://pubmed.ncbi.nlm.nih.gov/26113638/
  • Annu Rev Biochem. 2017 Jun 20; 86: 845–872. PMID: 28301742; https://pubmed.ncbi.nlm.nih.gov/30027111/
  • eNeuro. 2018 Jul 10;5(3). ENEURO.0174-18.2018. PMID: 30027111; https://pubmed.ncbi.nlm.nih.gov/28301742/
  • Appl. Sci. 2019, 9(4), 664; doi:10.3390/app9040664; https://www.mdpi.com/2076-3417/9/4/664
Patent Information:

The preceding is intended to be a non-confidential and limited description of a novel technology created at the University of Texas Health Science Center at Houston (UTHealth). This promotional material is not comprehensive in scope and should not replace company’s diligence in a thorough evaluation of the technology. Please contact the Office of Technology Management for more information regarding this technology.
For Information, Contact:
Yaya Lai
Technology License Associate
University of Texas Health Science Center At Houston
Chuan-Yar.Lai@uth.tmc.edu
Inventors:
John Spudich
Elena G Govorunova
Oleg A Sineshchekov
Keywords:
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