A team of researchers from the Duke Department of Surgery will be part of a multi-institutional research project funded by the U.S. Advanced Research Projects Agency for Health (ARPA-H) that will seek to advance sonogenetics for neuropathy treatment.
Aravind Asokan, PhD, Professor in Surgery, will lead the team at Duke under the Asokan Laboratory. Sreekanth Chalasani, PhD, of the Salk Institute, will serve as the project's principal investigator.
Sonogenetics is an emerging approach that sensitizes specific cell types to ultrasound by equipping them with ultrasound-responsive proteins, enabling precise, noninvasive control. The $41.3 million award will support a five-year, multi-institutional collaboration focused on developing core biological tools, next-generation ultrasound delivery systems, and the preclinical evidence needed to move sonogenetics into clinical trials for patients.
“We are rapidly entering an era of programmable medicines and this paradigm shift in treating patients in need will require thoughtful orchestration and translation of multimodal platform technologies into the clinic," says Dr. Asokan.
Utilizing mechano-sensitive proteins that can then be regulated by tools like ultrasound in order to alleviate neuropathy is one such example. This novel approach requires sustained but regulatable expression of these “sonochannels” in specific cellular subtypes, such as sensory neurons. The team has been developing engineered viruses for exactly these type of applications.
Engineered adeno-associated viral (AAV) vectors that enable safe and durable expression in neurological tissues provide the most promising path towards advancing gene therapies for rare, genetic disorders. The Asokan lab has been working for years on developing AAVs in translational animal models for clinical translation. The Duke Surgery team will combine this versatile platform with the power of regulatable gene expression and work with the Salk and SonoNeu teams to tackle chronic disease burden presented by peripheral neuropathies such as Diabetic Neuropathy or chronic inflammatory demyelinating polyneuropathy (CIDP) amongst others.
In addition to Duke, collaborating teams include: Scripps Research; Massachusetts Institute of Technology (MIT); University of California, San Diego; University of Manitoba; California Medical Innovations Institute (Calmi2); and SonoNeu.