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What is the new treatment for blindness?

Published in Gene Therapy for Blindness 2 mins read

A promising new treatment for inherited forms of blindness involves CRISPR-Cas9 gene editing, a revolutionary technology that offers the potential to restore vision by correcting genetic defects at their source.

Understanding CRISPR-Cas9 Gene Editing

CRISPR-Cas9 is a sophisticated molecular tool often described as a "GPS-guided scissor." Its primary function is to precisely target and cut specific sections of DNA.

  • Precision Targeting: Like a GPS, CRISPR-Cas9 can navigate to an exact location on the genome.
  • Gene Correction: Once at the target, it acts as a scissor, cutting out a portion of a mutated or faulty gene. This allows the cell's natural repair mechanisms to fill the gap with a functional, healthy gene.

Application for Inherited Blindness

For inherited blindness, the goal of CRISPR-Cas9 treatment is to address the underlying genetic mutations that prevent light-sensing cells in the eye from functioning correctly.

  1. Delivery Method: The CRISPR machinery is carefully injected into the eye, aiming to reach the retina. The retina is a light-sensitive layer at the back of the eye that contains specialized cells responsible for detecting light.
  2. Target Genes: The treatment targets specific mutated genes that are responsible for producing the proteins essential for these light-sensing cells.
  3. Restoring Function: By editing these mutated genes, the aim is to restore the eye's ability to produce the correct gene and protein, thereby allowing the light-sensing cells to function properly. This process has shown to lead to improvements in vision for individuals with inherited blindness.

This innovative approach offers a precise solution by directly mending the genetic errors that cause certain types of blindness, moving beyond symptomatic treatments to address the root cause of the condition.