May 3, 2024

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Gene Editing Technology Holds Promise to Eradicate HIV in the Future

CRISPR-Cas9 Gene Editing Technology EBT-001 Holds Promise to Eradicate HIV in the Future



 

CRISPR-Cas9 Gene Editing Technology EBT-001 Holds Promise to Eradicate HIV in the Future

August 18 – Scientists from Temple University’s Lewis Katz School of Medicine in the United States have recently reported that the CRISPR-Cas9 gene editing technology known as EBT-001 has the potential to safely and effectively remove SIV (Simian Immunodeficiency Virus) from the genomes of non-human primates.

This pre-clinical research represents a significant step forward in the pursuit of HIV therapy for humans, with the related paper published online in the “Gene Therapy” journal.

 

CRISPR-Cas9 Gene Editing Technology EBT-001 Holds Promise to Eradicate HIV in the Future

 

 

The research team, including Dr. Kamel Khalili, Dr. Tricia Burdo, and HIV researchers from Temple University, conducted experiments with EBT-001 in rhesus macaques, a tool specifically designed to target the SIV pre-viral DNA.

The findings demonstrate that EBT-001 efficiently eliminated SIV, which had been dormant in the viral reservoirs within the host’s DNA, with no off-target effects observed in the animals.

 

This novel technology aims to permanently inactivate the virus within large animal model tissues using a one-time injection treatment method, and the safety of this approach has been confirmed through these pre-clinical trials in non-human primates.

The team packaged the SIV-specific CRISPR-Cas9 gene editing construct, EBT-001, into an adeno-associated virus 9 (AAV9) vector, which can be delivered into the bodies of SIV-infected animals through intravenous injection.

 

This achievement lays the foundation for the ongoing EBT-101 clinical trials, marking not only a significant milestone in HIV virus research but also advancing the development of multiple gene editing therapies for other infectious diseases like herpes simplex virus and hepatitis B.

 

 

(source:internet, reference only)


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