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Gene addition for beta thalassemia

Janet L Kwiatkowski1,2

  • 1Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Annals of the New York Academy of Sciences
|October 13, 2023
PubMed

Individuals with transfusion-dependent beta thalassemia require a high burden of care and experience significant morbidity from the underlying disease and its treatment, which negatively impact the quality of life. Allogeneic hematopoietic stem cell transplantation offers the chance for a cure, but donor availability and transplant-related risks, especially in older patients, limit its use. Gene addition utilizing autologous CD34+ cells is an alternative, potentially curative, treatment option. Several clinical trials have investigated the use of lentiviral vectors containing a functional beta globin gene, including Lentiglobin BB305, GLOBE, and TNS9.3.55. The efficacy and safety data from these ongoing trials are discussed in this review.

Keywords:
beta thalassemiagene additiongene therapy

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