K. Lisa Yang and Hock E. Tan Center for Molecular Therapeutics in Neuroscience
The K. Lisa Yang and Hock E. Tan Center for Molecular Therapeutics in Neuroscience aims to change how we treat brain disorders by developing innovative molecular tools that precisely target dysfunctional genetic, molecular, and circuit pathways.
There are an estimated 19,000 to 22,000 genes in the human genome and a third of those genes are active in the brain–the highest proportion of genes expressed in any part of the body.
Variations in genetic code have been linked to many complex brain disorders, including depression and Parkinson’s. Emerging genetic technologies, such as the CRISPR gene editing platform pioneered by McGovern Investigator Feng Zhang, hold great potential in both targeting and fixing these errant genes. But the safe and effective delivery of this genetic cargo to the brain remains a challenge.
Researchers within the Yang-Tan Center for Molecular Therapeutics will improve and fine-tune CRISPR gene therapies and develop innovative ways of delivering gene therapy cargo into the brain and other organs. In addition, the center will leverage newly developed single cell analysis technologies that are revealing cellular targets for modulating brain functions with unprecedented precision, opening the door for noninvasive neuromodulation as well as the development of medicines. The center will also focus on developing novel engineering approaches to delivering small molecules and proteins from the bloodstream into the brain.
All tools developed within the center will be shared globally with academic and clinical researchers with the goal of bringing one or more novel molecular tools to human clinical trials.
Research at the center will initially focus on three major lines of investigation: genetic engineering using CRISPR tools, delivery of genetic and molecular cargo across the blood-brain barrier, and the development of novel tools to treat disorders of the nervous system in the clinical setting.
Our researchers are leading a broad effort to expand and improve the CRISPR gene editing platform to target genes associated with complex brain disorders. We are also developing advanced models and novel therapeutic approaches including RNA and DNA engineering for psychiatric and developmental disorders.
The CRISPR gene editing platform holds great potential in both targeting and fixing errant genes associated with brain disorders. But the safe and effective delivery of this genetic cargo to the brain remains a challenge. Our scientists will improve and fine-tune CRISPR gene therapies and develop innovative ways of delivering gene therapy cargo into brain tissue. We are also developing so-called “mini-genes,” small versions of genes that can be easily packaged and used to replace dysfunctional genes, and new delivery methods that are compatible with the human brain.
Our scientists work at the intersection of materials science, electronics, and neurobiology to design and fabricate optoelectronic and magnetic approaches to treat disorders of the nervous system. We are designing proteins that can cross the blood-brain barrier using primate cell models with the goal of moving these new molecular strategies into clinical trials in humans.