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