March 1, 2024

Lukmaan IAS

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THE EVOLUTION AND FUTURE OF CRISPR THERAPEUTICS

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TAG: GS 3: SCIENCE AND TECHNOLOGY

THE CONTEXT: The recent approvals by regulatory agencies in the UK and the US for CRISPR-based therapies targeting sickle-cell disease and β-thalassemia mark a transformative era in medicine.

EXPLANATION:

  • These inherited blood disorders affect millions globally, and the approvals signify a shift towards addressing their molecular basis rather than just symptom management.

THE EVOLUTION OF CRISPR TECHNOLOGY

  • Origins of CRISPR
    • CRISPR, an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, originated from the discovery of DNA elements in 1993 by Spanish researchers.
    • These elements were found in archaea and later in bacterial genomes.
    • Initially thought to be a part of the bacterial immune system against viruses, it was revealed that CRISPR, combined with CRISPR-associated proteins (Cas), functioned as an antiviral defense mechanism.
  • Milestone Discoveries
    • The groundbreaking work of Emmanuelle Charpentier, Jennifer Doudna, and Virginijus Siksnys led to key discoveries.
    • In 2010, it was demonstrated that CRISPR, specifically with Cas9 proteins, could cut DNA at precise points.
    • The identification of RNA molecules guiding Cas9 to specific genomic positions further revolutionized its potential.
    • This work culminated in the development of a programmable ‘molecular scissor’ capable of editing DNA accurately.
  • CRISPR-Cas9 Advancements
    • Subsequent research by Feng Zhang and George Church showcased CRISPR-Cas9’s ability to edit the genomes of eukaryotic organisms.
    • This innovation expanded its applications, ranging from genetic therapies to agricultural advancements.

CRISPR IN MEDICINE: CURRENT ACHIEVEMENTS AND FUTURE PROSPECTS

  • First-Generation Technologies
    • The approved CRISPR-based therapeutics represent first-generation technologies.
    • Though groundbreaking, they are continuously evolving to become more efficient and effective.
    • Novel approaches like base editing and prime editing hold immense promise for precise genome editing at the nucleotide level, addressing diseases like familial hypercholesterolemia.
  • Emerging Techniques and Challenges
    • Techniques like base editing and prime editing are showing potential, yet safety and accuracy issues persist.
    • Off-target events, where CRISPR systems inaccurately edit unintended parts of the genome, pose risks.
    • Balancing short-term benefits with long-term risks remains critical, especially as these therapies are still in early developmental stages.

THE FUTURE LANDSCAPE AND CONSIDERATIONS

  • Potential and Caution
    • While celebrating the transformative potential of CRISPR-based therapies like Casgevy, it’s essential to acknowledge potential risks.
    • Continued scrutiny and surveillance are imperative to identify and address unforeseen side effects.
    • The enormous promise of these technologies must be cautiously balanced with potential risks to ensure patient safety and ethical considerations.
  • Moving Forward
    • CRISPR technology has opened doors to a future where the correction of genetic anomalies is a reality.
    • The ongoing advancements in CRISPR-based therapies signify a promising era in medicine, holding immense potential to alleviate the suffering of millions affected by genetic diseases.

CONCLUSION

  • The approval of CRISPR-based therapies represents a significant leap in medical science, offering hope to patients with genetic disorders.
  • While these treatments mark a milestone, continuous research, vigilance, and technological advancements are crucial to maximize benefits while minimizing risks associated with genome editing technologies.

SOURCE: https://www.thehindu.com/sci-tech/science/crispr-casgevy-sickle-cell-disease-genetic-therapeutics-explained/article67641478.ece

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