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Mahesh

08/05/24 06:09 AM IST

CRISPR gene editing trial

In News
  • Scientists have said they used a human gene editing tool, CRISPR-Cas9, to restore vision in people with a rare form of inherited or congenital blindness.
CRISPR gene editing
  • The trial was called “BRILLIANCE” and 12 adults and two children, who had a rare form of inherited blindness, known as Leber congenital amaurosis (LCA), participated in it.
  • LCA affects about one in 40,000 people and causes severe vision loss at an early age.
  • This blindness is caused by a gene mutation that prevents a protein from functioning properly. That protein — CEP290 — is critical for sight.
  • Participants in the study received a single dose of a CRISPR gene therapy called EDIT-101.
  • CRISPR-Cas9 is a precise way of altering DNA. It cuts out specific strands of DNA — the thing that makes us who we are — and replaces them with a new strands.
  • In the case of EDIT-101, the treatment cuts out the mutation in CEP290 and inserts a healthy strand of DNA back into the gene.
  • This restores normal function of the protein CEP290, allowing the retina to detect light.
  • In 2020, Emmanuelle Charpentier and Jennifer A. Doudna were awarded the Nobel Prize in chemistry for discovering CRISPR-Cas9.
Significance
  • More than 200 people have been treated with experimental CRISPR technologies.
  • But so far, only one CRISPR treatment has been approved for clinical use — Casgevy, a treatment for sickle-cell disease — which has been available in the US, the UK, and the EU since December 2023.
  • Scientists have said they are entering a new phase in genome editing technologies, which they say they can safely help and cure — not just treat — patients with a variety of diseases.
  • Ongoing clinical trials are testing other CRISPR therapies for HIV/AIDS, diabetes, cancer, cardiovascular diseases, and antibiotic resistance.
Source- Indian Express

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