Headline: CRISPR Gene Editing Offers Hope for HIV Cure as Breakthrough Trial Shows Promising Results
Publication: Dailymail.com
Date: October 30, 2023
HIV, once a certain death sentence, may soon become a manageable chronic disease as gene editing experts make significant progress in finding a cure for the AIDS-causing virus. In a groundbreaking trial conducted in California, three patients have been injected with genetic material and an enzyme called CAS9, which has shown early potential in eliminating the sections of the HIV DNA lodged in human cells.
Using the revolutionary gene-editing technology called CRISPR, scientists are optimistic that a cure for HIV is now closer than ever before. While the current trial aims to establish the safety of the treatment, data on its efficacy is expected to be released next year. The new gene therapy utilizes an inactivated virus to deliver the gene editing tool to the body, specifically targeting immune cells that act as shields for HIV. Once attached to these cells, guide RNA guides the enzyme CAS9 to remove specific segments of the HIV genome, rendering the virus inactive.
HIV, which was once regarded as a near-certain death sentence, transformed into a chronic disease in the mid-90s with the introduction of antiviral medications. However, even with access to these medications, the risk of a dormant infection resurfacing and progressing to AIDS remains for the 1.2 million Americans living with HIV.
Over the years, treatment options for HIV have evolved significantly, from multiple daily pills to a single daily pill that combines all known therapies. These antiretroviral therapies are effective in suppressing the virus in the blood, but they are not a cure. The unique way in which HIV hijacks and hides in the body’s immune cells has made finding a cure challenging, as targeting the virus without damaging healthy cells is a complex task.
CRISPR technology shows exceptional promise in addressing chronic diseases like HIV due to its highly targeted nature. The therapy uses guide RNA to direct the enzyme CAS9 to specific targets on the DNA within cells, cutting the designated sections of the virus. Once the HIV cells are unable to replicate, they lose their ability to cause infection.
While CRISPR therapy holds great potential, its exorbitant cost could be a significant obstacle without insurance coverage. Novartis, a pharmaceutical company, launched a gene therapy for an inherited muscle wasting disease that costs $2.125 million per treatment. The researchers from Excision BioTherapeutics, a San Francisco-based biotech firm, developed the injectable gene therapy called EBT-101 for people with HIV, and the current trial has shown it to be safe.
The trial is still in its early stages, with only three participants having received the experimental treatment. These patients had stopped their antiretroviral medication beforehand and were injected with guide RNA and CAS9 to specifically target segments of the HIV DNA. The results from the trial are expected to be released next year, and six more individuals are set to participate.
The success of CRISPR technology extends beyond HIV, with breakthrough research into potential cures for sickle cell anemia and treatments for various types of cancer. CRISPR therapy has the potential to revolutionize the medical field by introducing targeted gene editing for numerous diseases.
Although a cure for HIV remains elusive, several individuals have been effectively cured, including the Berlin Patient (Timothy Ray Brown) and the London Patient (Adam Castillejo). The recent trial represents another important milestone in the fight against HIV, offering hope for a future without the burden of this devastating virus.
As the trial progresses, researchers aim to administer higher doses of the treatment and explore its potential efficacy on other chronic latent viral diseases beyond HIV, such as Herpes and Hepatitis B. The results of these endeavors will provide valuable insights into the future of gene therapies and the potential to eliminate the burden of infectious diseases for millions of people worldwide.