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CRISPR-Cas9 treats inherited blindness

Why is it in the news?

  • CRISPR-Cas9 has been employed to restore vision in individuals with a rare form of inherited or congenital blindness.

 More about the news

  • In the BRILLIANCE trial, individuals with Leber congenital amaurosis (LCA), a rare inherited blindness caused by mutations in the CEP290 gene, participated.
  • CRISPR-Cas9 gene editing technology was utilized to correct the genetic mutation, aiming to restore the function of the CEP290 protein critical for vision.

 About CRISPR-Cas9

  • CRISPR-Cas9 is a gene-editing technology derived from a bacterial defence system known as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated protein 9 (Cas9).
  • Cas9, the protein component of CRISPR-Cas9, acts as molecular scissors, capable of precisely cutting strands of DNA at specific locations determined by a guide RNA molecule.
  • CRISPR-Cas9 enables researchers to modify DNA sequences by either deleting, inserting, or altering specific genetic material within an organism’s genome.
  • This technology has numerous applications in various fields, including biomedicine, agriculture, and basic research. In medicine, it holds potential for treating genetic disorders, cancer, and infectious diseases.
  • In 2020, Emmanuelle Charpentier and Jennifer A. Doudna were awarded the Nobel Prize in Chemistry for their pioneering work in developing CRISPR-Cas9 as a gene-editing tool.
  • One of the key advantages of CRISPR-Cas9 is its high precision and efficiency in targeting specific DNA sequences, allowing for more accurate gene editing compared to previous methods.
  • However, the use of CRISPR-Cas9 in human germline editing raises significant ethical concerns regarding the potential for unintended consequences, the creation of designer babies, and widening societal inequalities.

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