March 27, 2014 | by Darrin Joy
John Rossi has been studying HIV — and how to help patients beat it — almost as long as scientists have known about the virus. His expertise is globally recognized, and it recently helped net him a five-year, $3.3 million grant from the National Institutes of Health (NIH).
The grant extends the NIH’s funding of Rossi’s work to more than two decades; it will support his efforts to develop a practical way to deliver a gene therapy to HIV patients.
The gene therapy uses a form of RNA, a close genetic cousin to DNA, to stop the virus from hijacking and destroying the immune system’s infection-fighting T cells. In fact, Rossi, who holds the Lidow Family Research Chair and is chair of the Department of Molecular and Cellular Biology, and his City of Hope colleagues have already conducted the only clinical trial to test this therapeutic RNA in patients.
Up until now, this treatment approach, called RNAi-based therapy, has only been available to HIV-positive patients with lymphoma who were undergoing stem cell transplantation. The transplant regimen is a necessary part of the process.
“For the vast majority of HIV patients to benefit from RNAi-based therapies, we need to find another way to deliver it to them that doesn’t require transplantation,” Rossi said.
Rossi and his team have identified three delivery methods that show promise for nontransplant patients, and the grant will help the scientists determine which, if any, will work and if the methods need further refinement.
Ultimately, Rossi hopes to find a way to deliver RNAi-based therapies that will fully protect HIV-positive patients from the virus, essentially curing them of disease.
Research reported in this publication was supported by the National Institute of Allergy and Infectious Diseases of the National Institutes of Health under grant number 2R01AI029329-24A1. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
Read more about John Rossi's research on the biology and application of small RNAs.