Light-gated Pentameric Channel - a New Optogenetic Tool

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent describes a newly identified light-gated pentameric ion channel, OLPVRII and its variants, derived from a viral rhodopsin. This channel can be genetically encoded and expressed in cells to control ion flow across membranes in response to light. It represents a next-generation optogenetic tool that enables precise and reversible activation or inhibition of neurons and other excitable cells using light, with potential for improved specificity and efficiency over existing channelrhodopsins. Genetic constructs, vectors, and methods for introducing and expressing these channels in various cell types are included, targeting both research and therapeutic uses.

Use CasesContent extracted from patent full text and abstract with AI.

• Restoring hearing by optogenetically stimulating auditory neurons in deaf patients, as a basis for advanced optical cochlear implants.
• Restoring vision in people with degenerative retinal diseases by making residual retinal cells light-responsive.
• Non-invasive, reversible control of brain circuits for research, mapping, and studying neural networks in neuroscience.
• Therapeutic intervention for neurological disorders such as Parkinson's and Alzheimer's, potentially modulating neuronal activity to alleviate symptoms or restore function.
• Treatment of seizures, brain injuries, or other neurodegenerative conditions through targeted neural modulation.
• Research tool for investigating the function of electrically excitable cells, including neurons, cardiac, or muscle cells, in vitro or ex vivo.
• Use in model organisms to study genetics of neural circuitry and behaviors.
• Ion transport studies or biotechnological applications in non-animal systems (e.g., plant, yeast, bacteria).

BenefitsContent extracted from patent full text and abstract with AI.

• Offers high light sensitivity and potentially larger photocurrents than current optogenetic tools, enabling better control over cell activity.
• Pentameric structure allows for unique and potentially more stable channel formation, with customizable ion selectivity (anion or cation).
• Can be genetically modified for improved properties or targeted to specific cell types, enhancing specificity of neural modulation.
• Human codon-optimized DNA sequences facilitate efficient expression in human and mammalian cells.
• Potential for reduced tissue invasiveness and improved spatial and temporal control due to optical activation.
• Broad therapeutic potential for restoring hearing, vision, or modulating neurological function in diseases.
• Versatile applications: suitable for in vitro, ex vivo, and in vivo studies.
• May enable higher resolution and more natural restoration of sensory functions compared to current electronic prostheses.

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Patent Abstract

The invention relates to newly characterized light-gated pentameric channels such as OLPVRII and their use in medicine, their utility as optogenetic tools, nucleic acid constructs encoding same, expression vectors carrying the nucleic acid construct, cells expressing said light-gated pentameric channels and their respective uses.