ESGCT 2021 | Gene therapy for PKD: addressing the unmet clinical need

Jose-Luis López-Lorenzo, MD, University Hospital Jimenez Diaz, Madrid, Spain, describes the therapeutic landscape for pyruvate kinase deficiency (PKD), a rare inherited hemolytic anemia caused by mutations in the PKLR gene. Treatment of PKD is currently largely limited to blood transfusion, chelation therapy and splenectomy, which are associated with significant side effects. Dr López-Lorenzo discusses the rationale behind the development of gene therapies for PKD, such as the transplantation of autologous hematopoietic stem cells containing a functional copy of the deficient PKLR gene. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.