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Journal Article Review Epublish 28 citations

PubMed: 34200975 DOI: 10.3390/ijms22126275

Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing.

Int J Mol Sci 22(12)

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

Affiliations

  • International PhD Program for Cell Therapy and Regeneration Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan.
  • Department of Biochemistry and Molecular Cell Biology, School of Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan.
  • Faculty of Medicine and Health Sciences, Universitas Muhammadiyah Makassar, Makassar 90221, Indonesia.
  • International PhD Program for Cell Therapy and Regeneration Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan. Department of Biochemistry and Molecular Cell Biology, School of Medicine, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan.
  • Research Center of Cell Therapy and Regeneration Medicine, Taipei Medical University, Taipei 11031, Taiwan.
  • Graduate Institute of Medical Sciences, College of Medicine, Taipei Medical University, Taipei 11031, Taiwan.
  • Center for Reproductive Medicine, Taipei Medical University Hospital, Taipei 11031, Taiwan.
  • Comprehensive Cancer Center, Taipei Medical University, Taipei 11031, Taiwan.
  • Research Center of Cancer Translational Medicine, Taipei Medical University, Taipei 11031, Taiwan.
  • PhD Program for Translational Medicine, College of Medical Science and Technology, Taipei Medical University, Taipei 11031, Taiwan.
  • Department of Histology, Faculty of Medicine, Universitas Indonesia, Jakarta 10430, Indonesia.
  • Stem Cell Medical Technology Integrated Service Unit, Cipto Mangunkusumo Central Hospital, Faculty of Medicine, Universitas Indonesia, Jakarta 10430, Indonesia.
  • Stem Cell and Tissue Engineering Research Center, Indonesia Medical Education and Research Institute (IMERI), Faculty of Medicine, Universitas Indonesia, Jakarta 10430, Indonesia.

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 endeavors 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.

MeSH

Thalassemia, Transplantation, Anemia, Therapeutics, Microscopy, Electron, Scanning Transmission, Glucosephosphate Dehydrogenase Deficiency, Genes, Hematopoietic Stem Cells, Glucose-6-Phosphate, Gene Editing

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