CRISPR Gene Editing Offers Hope for Curing HIV
HIV, which was once a death sentence, may soon become a curable disease thanks to breakthroughs in gene editing. Researchers have injected three patients in California with genetic material and an enzyme called CAS9, which has the potential to eliminate sections of HIV DNA from human cells. This treatment, utilizing the CRISPR gene-editing technology, brings us closer than ever before to finding a cure for AIDS.
The ongoing trial is primarily focused on proving the safety of the treatment, with efficacy data expected to be released next year. The gene therapy works by delivering the gene editing tool to immune cells using an inactivated virus. Guide RNA then guides the enzyme CAS9 to specific segments of the HIV genome, effectively removing the virus and rendering it inactive.
Antiviral medications turned HIV into a chronic disease rather than a death sentence in the mid-90s, but approximately 1.2 million Americans still live with the virus. Even with access to medication, there is a risk of the dormant infection resurfacing and progressing to AIDS. Current treatments, such as antiretroviral therapies, can suppress the virus but are not a cure. The unique way in which HIV hijacks the body’s cells has made finding a cure challenging. However, CRISPR technology offers hope due to its precise targeting of specific sections of genetic material within cells.
While the potential of CRISPR therapy is highly promising, it is worth noting that the high cost of the treatment could limit its accessibility. Similar gene therapies have been launched with exorbitant price tags, such as Novartis’ treatment for an inherited muscle wasting disease that costs $2.125 million per treatment. Nevertheless, exciting progress has been made, with the experimental CRISPR therapy showing safety and efficacy in early stages. The therapy has been injected into three patients with HIV so far and has shown no severe negative side effects.
Researchers from Excision BioTherapeutics are encouraged by the initial results and plan to trial higher doses of the gene therapy. Their goal is to treat chronic latent viral diseases beyond HIV, potentially bringing us closer to a cure for other diseases like Herpes and Hepatitis B. The field of CRISPR gene editing has also shown promise in treating sickle cell anemia and certain types of cancer.
Although there have been a few cases of individuals effectively cured of HIV through stem cell transplants, gene editing offers a less risky and more accessible solution. The Dusseldorf Patient, a 53-year-old man, has been off antiretroviral drugs for four years without relapse after undergoing an allogeneic hematopoietic stem cell transplantation. Similarly, Timothy Ray Brown, also known as the Berlin patient, was declared HIV-free in 2007 after receiving two bone marrow transplants.
Overall, the progress made in CRISPR gene editing brings hope to the millions of individuals living with HIV. While there are still challenges to overcome, the potential to cure HIV and other chronic diseases is within reach.
