Cell and gene therapy for anemia: Hematopoietic stem cells and gene editing

Dito Anurogo, Nova Yuli Prasetyo Budi, Mai Huong Thi Ngo, Yen Hua Huang, Jeanne Adiwinata Pawitan

Research output: Contribution to journalArticlepeer-review

1 Citation (Scopus)

Abstract

Hereditary anemia has various manifestations, such as sickle cell disease (SCD), Fanconi anemia, glucose‐6‐phosphate dehydrogenase deficiency (G6PDD), and thalassemia. The available management strategies for these disorders are still unsatisfactory and do not eliminate the main causes. As genetic aberrations are the main causes of all forms of hereditary anemia, the optimal approach involves repairing the defective gene, possibly through the transplantation of normal hematopoietic stem cells (HSCs) from a normal matching donor or through gene therapy approaches (either in vivo or ex vivo) to correct the patient’s HSCs. To clearly illustrate the importance of cell and gene therapy in hereditary anemia, this paper provides a review of the genetic aberration, epidemiology, clinical features, current management, and cell and gene therapy en-deavors related to SCD, thalassemia, Fanconi anemia, and G6PDD. Moreover, we expound the future research direction of HSC derivation from induced pluripotent stem cells (iPSCs), strategies to edit HSCs, gene therapy risk mitigation, and their clinical perspectives. In conclusion, gene‐corrected hematopoietic stem cell transplantation has promising outcomes for SCD, Fanconi anemia, and thalassemia, and it may overcome the limitation of the source of allogenic bone marrow transplantation.

Original languageEnglish
Article number6275
JournalInternational Journal of Molecular Sciences
Volume22
Issue number12
DOIs
Publication statusPublished - 2 Jun 2021

Keywords

  • Anemia
  • Cell therapy
  • Gene editing
  • Gene therapy
  • Hematopoietic stem cells

Fingerprint

Dive into the research topics of 'Cell and gene therapy for anemia: Hematopoietic stem cells and gene editing'. Together they form a unique fingerprint.

Cite this