Paper: GS – III, Subject: Science and Technology, Topic: Biotechnology, Issue: CRISPR-Cas9 Gene Editing.
Context:
The groundbreaking case of Kyle “KJ” Muldoon Jr., a nine-month-old boy who became the first known recipient of a custom (CRISPR-Cas9) gene-editing treatment for a rare genetic disorder.
Key Takeaways:
About CRISPR-Cas9:
- CRISPR/Cas9, short for Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technology that can be used to alter the genetic sequence of a specific gene by removing, adding, or altering sections of the DNA sequence.
- It makes it possible to correct errors in the genome and turn on or off genes in cells and organisms quickly, cheaply, and with relative ease.
- Mechanism: When a virus infects a bacterial cell, the bacterium captures a piece of the virus’s genome and incorporates it into its own DNA.
- This allows the bacterium to produce a guide RNA that can recognize the virus in future infections.
- Functionality: The guide RNA directs an enzyme called Cas9 to cut the viral DNA, effectively neutralizing the threat.
CRISPR/Cas9 the ABCs of DNA:
How Does CRISPR-Cas9 Gene Editing Work?
- Identification: The first step involves locating the abnormal DNA sequence responsible for a genetic disorder.
- Guide RNA Creation: Scientists create a guide RNA that matches the target DNA sequence.
- Cutting the DNA: The guide RNA directs the Cas9 enzyme to make a double-strand break in the DNA at the specified location.
- Repair Process: The DNA strands naturally attempt to repair themselves, but scientists can introduce a correct DNA sequence to replace the faulty one.
The Case of KJ Muldoon Jr. Personalized Treatment:
- Scientists identified the specific mispaired base causing KJ’s CPS1 deficiency and programmed the base editing tool to correct it.
- Advantages: Base editing offers a more straightforward approach compared to traditional CRISPR methods, reducing the need for foreign DNA insertion.
- Hope for Rare Disorders: KJ’s successful treatment has sparked optimism for other patients with rare genetic disorders.
CRISPR/Cas9 Challenges:
- Ahead Cost: The treatment for KJ was likely funded by research institutes and biotechnology firms, with costs potentially reaching hundreds of thousands of dollars.
- Scalability: The personalized nature of KJ’s treatment means it cannot be easily replicated for others with different disorders, posing challenges for mass production.
- Regulatory Hurdles: Gaining regulatory approvals for such treatments can be complex and time-consuming, particularly in countries with stringent regulations.
Conclusion:
- While the successful application of base editing in KJ Muldoon Jr.’s case represents a significant advancement in gene therapy, numerous challenges remain before such treatments can become widely accessible.
- The high costs, the need for personalized solutions, and regulatory complexities must be addressed to ensure that more patients can benefit from these revolutionary technologies.
- Until then, only a fortunate few will have access to the potential of base editing therapies.
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