Gene Therapies for Transfusion-Dependent b-Thalassemia.

Abstract

b-Thalassemia is one of the most prevalent monogenic diseases usually caused by quantitative defects in the production of b-globin, a component of adult hemoglobin (a2b2), leading to severe anemia. Technological advances in genome sequencing, stem cell selection, viral vector development, transduction and gene-editing strategies now allow for efficient ex-vivo genetic manipulation of human hematopoietic stem cells that can lead to a meaningful clinical benefit in thalassemia patients. In this perspective, the status of the gene-therapy approaches available for transfusion-dependent thalassemia and early results of clinical trials are discussed. It is highly anticipated that gene therapies will soon become a treatment option for patients lacking compatible donors for hematopoietic stem cell transplant and will offer a suitable alternative for definitive treatment of b-thalassemia, even in young children.

• Authors: Sandeep Soni
• Journal: Indian pediatrics (Indian Pediatr) Vol. 58 Issue 7 Pg. 667-674 (Jul 15 2021) ISSN: 0974-7559 [Electronic] India
• PMID: 33772535 (Publication Type: Journal Article)

Chemical References

• Hemoglobins

Topics

• Child, Preschool
• Genetic Therapy
• Genetic Vectors
• Hemoglobins
• Humans
• Thalassemia
• beta-Thalassemia (genetics, therapy)