Translational Neuroscience Research Group

UC San Diego Neurosciences has gained an international reputation as a leader in translational medicine — bridging the gap between research and treatment.

Contributions to Therapies

Our researchers have directly contributed to the development of now-approved therapies for neurological disease, including:

• rt-PA for stroke
• Nneuroprotective therapies for Parkinson’s disease
• Cholinesterase inhibitors for Alzheimer’s disease
• Vagal nerve stimulation for epilepsy

Therapies currently undergoing translational trials include:

• Anti-inflammatory drugs for Alzheimer’s disease
• Growth factor gene therapy for Alzheimer’s disease, Parkinson’s disease, and ALS
• Cannabinoids for spasticity

The Challenge

There has never been a more exciting time to explore, understand and treat disorders of the nervous system.

The tools now available to understand neurological disorders offer an unprecedented opportunity to identify disease targets and develop and test novel therapies that could revolutionize treatment.

But to bring this revolution to light, we need to build on our translational programs to close the gap between discoveries made in the research lab and clinical treatments for patients.

The Innovation

A new Translational Neuroscience Institute (TNI) at UC San Diego will combine disciplines to bring advances in basic neuroscience from early discovery to human clinical trials. We will be able to identify and develop new therapies as this approach illuminates our knowledge of disease mechanisms.

UC San Diego and the other neuroscience programs in La Jolla represent what may be the world’s most advanced concentration of scientific and clinical knowledge for translational neuroscience. We plan to leverage this community’s successful track record in translation of drug, cell and gene therapies.

Translational Neuroscience Institute (TNI) Components

Preclinical discovery

• Discovery of disease mechanism: investigate cell-specific disease mechanism
• Proteomics:
  • Identify protein mechanisms of disease and targets
  • Develop specific neuroscience expertise in proteomics in collaboration with other campus proteomics programs
• Lipidomics: characterize the lipid state of the cell and potential new disease targets
• Bioinformatics: tie it all together
  • Develop specific neuroscience expertise in bioinformatics
  • Prioritize targets from extensive data sets

Pre-clinical development

• Develop novel therapeutics
  • Develop in vitro assays and high throughput
  • Utilize high throughput screening capabilities, including NIH Blueprint for Neuroscience Research
  • Parallel effort: Develop novel therapeutic technologies:
    • Gene therapy, cell therapy, bioengineering

Clinical translation

• Establish a Clinical Translation Core to design and perform human trials using medical and surgical specialties

Education and training

• Educate and Train translational scientists and clinicians
  • Offer training fellowships
  • Sponsor weekly informal lab meetings, monthly lecture series, and an annual translational neuroscience seminar