2023-08-21 12:45:18
A new CRISPR gene-editing treatment, delivered via a single injection, safely and efficiently removes SIV (simian immunodeficiency virus), a virus related to the virus that causes AIDS, from the genomes of non-human primates .
This has been verified by a team headed by Tricia H. Burdo from Temple University in the US city of Philadelphia.
The achievement complements previous experiments as the basis for the first clinical trial of a gene-editing technology against HIV in human patients, which was cleared by the US Food and Drug Administration (FDA) in 2022.
The preclinical study tested EBT-001, an SIV-specific CRISPR-Cas9 gene-editing therapy, in rhesus macaque monkeys.
The study shows that EBT-001 effectively removes SIV from reservoirs (cells and tissues where viruses such as SIV and HIV integrate into the host’s DNA and hide for years) with no detectable side effects in animals.
Analyzes show that the action of EBT-001 was widespread, reaching tissues throughout the body, with evidence of gene editing of SIV proviral DNA in all significant viral reservoirs.
Image captured with a scanning electron microscope, and later reprocessed, in which the spherical green particles are AIDS viruses infecting a human cell. (Image: CDC/C. Goldsmith/P. Feorino/EL Palmer/WR McManus)
Furthermore, EBT-001 was well tolerated at all dose levels, with no evidence of toxicity on clinical examination of the animals or after histopathological investigation. Even with the naked eye the positive effect was evident. “The treated animals looked healthier and some gained weight,” explains Kamel Khalili, from Temple University and a member of the research team.
This advance is a big step towards obtaining the definitive cure for AIDS in humans.
The study is titled “Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates”. And it has been published in the academic journal Gene Therapy. (Source: NCYT from Amazings)
#eliminate #AIDS #virus #gene #editing