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Gene editing shows promise as curative treatment for HIV


Antiretrovirals have done a good job in halting the progression of HIV to AIDS, but new gene editing technologies could result in a cure.

Antiretroviral therapy has changed the face of HIV/AIDS treatment, but new research in gene editing could be curative, according to a new report.

Kamel Khalili, PhD, Laurel H. Carnell professor and chair in the department of neuroscience, director of the Center for Neurovirology and director of the Comprehensive NeuroAIDS Center at Temple University in Philadelphia, said gene editing could eliminate HIV infection in cells completely. Current treatment of HIV with antiretroviral therapy (ART) has been successful in suppressing viral replication, said Khalili, but it’s not a perfect treatment. There are several issues with ART including cost, compliance, co-morbidities, and the development of resistant virus.

“Importantly, the current ART is not designed to eliminate the virus so the virus will return if patients stop treatment,” Khalili told Medical Economics. “The gene editing technology which we have developed is designed to completely and permanently eliminate the HIV-1 virus from infected cells and thus, this strategy is a cure.”

ART was developed in the 1990s and has been successful in making HIV a chronic condition that now rarely progresses to the development of AIDS. However, Khalili’s research notes that this treatment requires lifelong medication that can have a negative impact on other body systems. Advances in gene editing show promise, through, and have allowed researchers to inactivate integrated proviral DNA in the genome of latently infected cells or eliminate HIV receptors. Several trials are being performed on variety of gene editing methods, but challenges include the generation of viral escape mutants, avoidance unintended effects, and how to best deliver the reagents to HIV-infected cells in patients, the study notes.

Next: The potential of CRISPR


‘Enormous potential’ of CRISPR

Khalili’s paper reviews several different methods, and earlier this year his team used a gene editing method called CRISPR to eliminate HIV infection in mice. CRISPR stands for clustered regularly interspaced short palindromic repeats. The gene editing system can target specific pieces of genetic code to edit DNA or RNA resulting in permanent modifications to treat genetic abnormalities and disease. Researchers are now working to secure funding to continue studies in primates.

“The CRISPR gene editing technology has enormous potential in the clinic, not only for infectious diseases such as HIV-1, but for many other illnesses including cancer and genetic disorders as well,” Khalili said.

Additional clinical trial success is needed to make this kind of treatment available in clinical practice, but Khalili said he is hopeful that advanced CRISPR results in HIV treatment will be available within the next three years.

“Our gene editing approach is to eliminate the virus and thus eliminate the disease, taking the patient from a chronic long term infection to a disease-free state,” Khalili said. “We want clinicians to see that the cure of AIDS may be possible.”

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