First Patient Begins Newly Approved Sickle Cell Gene Therapy

• Commercially approved gene therapy can now cure the condition.

Gene therapy

• It is a technique that involves altering the genetic material of cells to treat or prevent disease.
• It aims to introduce a normal, functional gene to compensate for the defective, disease-causing gene.
• It employs various approaches: replacing a mutated gene with a healthy copy, inactivating a mutated gene, and introducing a new gene.
• Unlike traditional drugs, gene therapy targets the root genetic causes inside cells.
• Active clinical trials approach various inherited and acquired disorders.
• Approaches include ex vivo modification of hematologic stem cells, T lymphocytes, and other immune cells, and in vivo delivery of genes or gene editing reagents to relevant target cells.

Prelims Takeaway:

• Gene therapy
• Sickle cell disease