Allogeneic hematopoietic stem cell transplantation for inherited diseases

Allogeneic hematopoietic stem cell transplantation (HSCT) has been applied for the treatment of inherited bone marrow failure syndromes, inherited metabolic diseases, and primary immunodeficiencies. HSCT indications for some inherited disorders are still controversial because the effects on neurocognitive function have been found to be variable, according to pre-transplant conditions including the patient's age, disease severity, disease status, and donor availability. Moreover, utmost attention must be paid to determining the preparative regimen for preventing regimen-related toxicity because the genetic effect was found to be expressed not only in the hematopoietic stem cells themselves but also in systemic tissues. Herein, recent progress and advances in the fields of HSCT for inherited diseases are reviewed.