On gene therapy – Game-changer

Source: This post on gene therapy is created based on the article “game changer”, published in The Hindu on 13^th December 2023.

Syllabus Topic: GS Paper 3 – Science and technology

News: Recently, the UK drug regulator and the U.S. FDA approved the gene therapies Casgevy and Lyfgenia for treating sickle cell disease in patients over 12 years.

This marks a significant advancement in gene therapy using the CRISPR-Cas9 tool for diseases traditionally treated through bone marrow transplantation.

Read more about the technology

What are the advantages of Using CRISPR-Cas9?

Patient’s Own Blood Cells: The use of the patient’s own blood cells for gene editing is a major advantage, potentially allowing treatment for a large number of patients without the need for donor matching.

Innovative Approach: The application of CRISPR-Cas9 in these therapies is a significant leap forward in genetic medicine, offering hope for many hereditary conditions.

What are the challenges and considerations of using the CRISPR-Cas9?

Cost and Accessibility: Despite the potential, these treatments are expected to be extremely expensive, limiting their accessibility.

Hospital Capabilities: Only certain hospitals equipped with specific technologies can administer these therapies, further restricting access.

Safety and Efficacy Monitoring: Continuous monitoring for unintended genetic modifications and side effects is crucial due to the novel nature of these therapies.