Publikasi Scopus FKUI 2021 per tanggal 30 Juni 2021 (428 artikel)

Publikasi Scopus FKUI 2021 per tanggal 30 Juni 2021 (428 artikel)

Anurogo D., Budi N.Y.P., Ngo M.-H.T., Huang Y.-H., Pawitan J.A.
57224408026;57200656694;57222023578;57154840200;6508348067;
Cell and gene therapy for anemia: Hematopoietic stem cells and gene editing
2021
International Journal of Molecular Sciences
22
12
6275
https://www.scopus.com/inward/record.uri?eid=2-s2.0-85107482996&doi=10.3390%2fijms22126275&partnerID=40&md5=fea16158471961b74a29fcf9cb158461
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
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
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.
Anemia; Cell therapy; Gene editing; Gene therapy; Hematopoietic stem cells
MDPI AG
16616596
Article
Q1
1317
2808

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