• UGI USER
  • Dec 19, 2023
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CRISPR THERAPEUTICS: REVOLUTIONIZING GENETIC MEDICINE

Why in the News ?

  • Global Recognition: UK’s Medicines and Healthcare products Regulatory Agency approved exagamglogene autotemcel for sickle-cell disease and ß-thalassemia.
  • Dual Approval: S. FDA also sanctioned it for sickle-cell disease, making it a pioneering CRISPR-based therapeutic approved by two major regulators.
Source: Science in News

About CRISPR technology:

  • Overview: CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene editing technology inspired by bacteria’s natural defence mechanism against viruses, utilizing the Cas9 protein.
  • Genetic Engineering Process: CRISPR involves manipulating genes, either adding new ones or suppressing existing ones, constituting a form of genetic engineering.
  • No External Gene Introduction: Unlike some genetic engineering methods, CRISPR doesn’t introduce external genes but works with the organism’s existing genetic material through the CRISPR-Cas9 system.
  • CRISPR-Cas9 as ‘Genetic Scissors’: CRISPR-Cas9 is often referred to as ‘Genetic Scissors’ for its precision in cutting and modifying genetic material, making it a powerful tool in genetic manipulation.

 CRISPR Evolution:

  • Three-Decade Journey: CRISPR discovery resulted from 30 years of academic pursuit, evolving from DNA elements in archaea to a powerful genome-editing tool.
  • Molecular Scissors: CRISPR-Cas9 system emerged as a programmable ‘molecular scissor’ in 2012, allowing precise editing of DNA.

Medical Milestones:

  • Casgevy Approval: UK and US approvals for Casgevy mark a monumental leap in treating sickle-cell disease at its molecular basis.
  • First-Generation Technologies: While ground breaking, these approvals represent first-generation CRISPR technologies.

Advanced Approaches:

  • Base Editing: Cutting-edge base-editing refines genome editing at a single nucleotide level.
  • Prime Editing: Researchers explore a ‘search-and-replace’ strategy for accurate sequence insertion.
  • Epigenetic Modification: CRISPR applications extend to modifying epigenetic effects in a targeted manner.

Future Considerations:

  • Safety Challenges: Concerns include off-target CRISPR-Cas9 events, requiring a balance between risks and benefits.
  • Technological Scrutiny: Ongoing surveillance will uncover potential side effects as these therapies advance.

Optimistic Outlook:

  • Patient Impact: Approvals like Casgevy offer hope for millions with genetic diseases, signalling a promising era in genetic medicine.
  • Continuous Advancements: Despite challenges, the future of CRISPR technologies holds tremendous promise for genetic engineering and medical advancements.