Publikasi Scopus 926 artikel (Per 14 Maret 2022)

Riset dan Pengabdian Masyarakat Do Research Sosial Service and Innovation

Publikasi Scopus 926 artikel (Per 14 Maret 2022)

335

Authors

Anurogo D., Budi N.Y.P., Ngo M.-H.T., Huang Y.-H., Pawitan J.A.

Author S Id

57224408026;57200656694;57222023578;57154840200;6508348067;

Title

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

Year

2021

Source Title

International Journal of Molecular Sciences

Volume

Issue

Art No

6275

Page Start

Page End

Page Count

Cited By

Link

https://www.scopus.com/inward/record.uri?eid=2-s2.0-85107482996&doi=10.3390%2fijms22126275&partnerID=40&md5=fea16158471961b74a29fcf9cb158461

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; 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

Authors With Affiliations

Anurogo, D., 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; Budi, N.Y.P., 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; Ngo, M.-H.T., 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; Huang, Y.-H., 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; Pawitan, J.A., 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 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. © 2021 by the authors. Licensee MDPI, Basel, Switzerland.

Author Keywords

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

Index Keywords

antisickling agent; BRCA1 protein; BRCA2 protein; Fanconi anemia group A protein; Fanconi anemia group b protein; Fanconi anemia group C protein; Fanconi anemia group D2 protein; Fanconi anemia group E protein; Fanconi anemia group F protein; Fanconi anemia group G protein; Fanconi anemia group i protein; Fanconi anemia group j protein; Fanconi anemia group L protein; hematologic agent; hemoglobin beta chain; hemoglobin F; nuclear protein; partner and localizer of BRCA2; Rad51 protein; Rad51c protein; unclassified drug; acute chest syndrome; anemia; aplastic anemia; arm malformation; Article; asplenia; blood transfusion; cafe au lait spot; cell therapy; cerebrovascular accident; chromosome aberration; chronic nonspherocytic hemolytic anemia; clinical feature; clinical outcome; congenital d

Publisher

MDPI

Issn

16616596

Isbn

Pubmed Id

34200975

Document Type

Article

Sjr Best Quartile

Sjr

1455

Sjr Rank

2384