Human papillomavirus

Novel CRISPR Gene-Editing Treatment for HIV- Virus.

In a groundbreaking advancement, researchers at the Lewis Katz School of Medicine at Temple University have successfully developed and tested a revolutionary gene-editing treatment capable of eliminating a virus related to HIV from the genomes of non-human primates. The study, published in the journal Gene Therapy, introduces a novel CRISPR-Cas9 gene-editing therapy known as EBT-001, which specifically targets the Simian Immunodeficiency Virus (SIV), a close relative of the virus responsible for causing AIDS in humans.

The research demonstrates the efficacy and safety of EBT-001 by effectively removing SIV from viral reservoirs within the primates’ cells and tissues. Unlike traditional treatments, EBT-001 showcases precision gene-editing capabilities without causing any detectable off-target effects, ensuring a high degree of accuracy and safety.

This breakthrough paves the way for the first-ever clinical trial of an HIV gene-editing technology in human patients, authorized by the Food and Drug Administration (FDA) in 2022. The innovative treatment is the result of collaborative efforts between researchers at the Lewis Katz School of Medicine and scientists at Excision BioTherapeutics, Inc.

To ensure safety and effectiveness, the researchers conducted meticulous preclinical trials using non-human primates. The study involved adapting EBT-101 to treat SIV infection, a virus that closely mimics HIV infection in humans. EBT-001 was delivered via an adeno-associated virus 9 (AAV9) carrier through intravenous injections.

The outcomes of the study were promising. EBT-001 was distributed broadly throughout the primates’ bodies, successfully editing SIV proviral DNA within significant viral reservoirs. Importantly, the treatment exhibited excellent tolerability across various dosage levels, with no evidence of toxicity.

This achievement holds immense promise not only for advancing the potential cure for HIV/AIDS but also for inspiring multiplex gene editing therapies targeting other infectious diseases. The successful preclinical model sets the stage for Excision BioTherapeutics’ ongoing clinical trial program, which aims to evaluate the safety and efficacy of EBT-101 in human patients.

Jennifer Gordon, Senior Vice President of Research and Development at Excision BioTherapeutics, highlights the importance of this study in paving the way for clinical trials that could potentially lead to a cure for people living with HIV. With the authorization for clinical trials and the promising results from the preclinical study, researchers and the medical community are excited about the transformative potential of this gene-editing treatment.

The groundbreaking research signifies a significant step forward in the pursuit of effective HIV/AIDS treatments and has the potential to revolutionize gene therapy approaches for various infectious diseases. As the clinical trials progress, the medical world eagerly anticipates the outcomes of this innovative gene-editing technology.


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