The National Institutes of Health (NIH) has awarded three labs from the University of Pennsylvania (UPenn; Philadelphia, PA) $12.5 million worth in grants to help innovate and speed medical research, including work involving optogenetics, microscopy, and fluorescent probes. All part of the NIH’s $143.8 million national grant program, these awards are granted under the NIH Director’s Pioneer, New Innovator, and Transformative Research Projects Awards.
Key investigators on the Pioneer Award are Jean Bennett, MD, Ph.D., principal investigator of the study; Luk Vandenberghe, Ph.D.; and Albert M. Maguire, MD, all at UPenn's F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, Perelman School of Medicine. The team has been awarded $4 million over the next five years to use optogenetics—a type of gene therapy—to treat inherited forms of blindness, which can be caused by mutations in any of hundreds of different genes. Optogenetics in this case involves re-sensitizing the blind eye by delivering light-sensitive molecules to the remaining retinal cells. The team's study aims ultimately to test the safety and efficacy of optogenetics in blind patients in the clinic, which could pave the way for development of novel gene therapy approaches for the treatment of other devastating sensory diseases.
Arjun Raj, Ph.D., assistant professor of Bioengineering, School of Engineering and Applied Science, received the New Innovator Award for $1.5 million over five years. His research involves developing and applying new microscopic imaging tools to reveal how the physical organization of the genetic code determines the manner in which the cell reads the code itself. The development of these methods will allow researchers to directly visualize genetic organization in single cells. An understanding of this organization will be important for elucidating how defects in translating the genetic code contribute to such diseases as cancer.
A $7 million, five-year Transformative Research Project Award was given to a team of researchers from the Perelman School of Medicine, Emory University, and Georgia Tech, including Sunil Singhal, MD, director of the Thoracic Surgery Research Laboratory at UPenn. Seeking a method to make tumors more visible and easier to distinguish from surrounding tissues in order to remove them completely, the team has developed fluorescent nanoparticle probes that find and focus on cancer cells. The researchers’ main goals are to help surgeons distinguish tumor edges; identify diseased lymph nodes and to determine if the tumor has been completely removed. Having these capabilities can be expected to make a major impact in reducing recurrence rates of lung cancer after surgery. The grant includes plans for tests of the nanoparticles in animal models and a first-in-human clinical trial for patients with lung cancer. The proposed technologies could be broadly applicable to many types of solid tumors.
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