The era of CRISPR therapeutics is here – what can we expect?
- December 18, 2023
- Posted by: OptimizeIAS Team
- Category: DPN Topics
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The era of CRISPR therapeutics is here – what can we expect?
Subject :Science and Tech
Section: Biotechnology
Context:
- The medical world is witnessing a significant breakthrough with the approval of CRISPR-based therapies for sickle-cell disease and β-thalassemia in the U.K. and the US.
- These advancements hold the potential to transform the lives of millions suffering from these inherited blood disorders.
Historical Background of CRISPR Technology
- Origins of CRISPR: Discovered in archaea in 1993, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) elements were later found to form an antiviral defense system in bacteria with Cas (CRISPR-associated) proteins.
- Nobel Prize-Winning Innovation: Emmanuelle Charpentier and Jennifer Doudna’s work on CRISPR-Cas9 as a ‘molecular scissor’ earned them the 2020 Nobel Prize in chemistry.
- Eukaryotic Genome Editing: Subsequent research demonstrated CRISPR-Cas9’s ability to edit eukaryotic genomes, paving the way for various applications in genetic therapies and agriculture.
CRISPR Applications in Medicine
- CRISPR-Based Treatment for Blood Disorders: The MHRA in the U.K. and the FDA in the U.S. approved ‘Casgevy’ for treating sickle-cell disease and transfusion-dependent β-thalassemia.
- Treatment Mechanism: Casgevy involves modifying a patient’s blood stem cells to correct the genetic defect causing sickling, then regrafting them to produce normal red blood cells.
- Historical Context: This approval marks a full circle from Linus Carl Pauling’s description of sickle-cell disease as a molecular disorder 74 years ago.
Emerging CRISPR Technologies and Approaches
- Base-Editing: This technique allows genome editing at the single nucleotide level.
- Prime Editing: A newer method that uses a search-and-replace strategy for precise genome modifications.
- Epigenetic Modifications: CRISPR systems are also being developed to target epigenetic effects.
Future Prospects and Challenges
- Safety and Accuracy Concerns: Issues like off-target events, where CRISPR-Cas9 edits unintended parts of the genome, pose significant challenges.
- Balancing Risks and Benefits: While the potential of these technologies is enormous, their risks must be weighed against both short- and long-term benefits.
- Ongoing Research and Surveillance: Continuous scrutiny is essential to uncover potential side effects that are currently unknown.