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

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 ab...

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Autores principales: Anurogo, Dito, Yuli Prasetyo Budi, Nova, Thi Ngo, Mai-Huong, Huang, Yen-Hua, Pawitan, Jeanne Adiwinata
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8230702/
https://www.ncbi.nlm.nih.gov/pubmed/34200975
http://dx.doi.org/10.3390/ijms22126275
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author Anurogo, Dito
Yuli Prasetyo Budi, Nova
Thi Ngo, Mai-Huong
Huang, Yen-Hua
Pawitan, Jeanne Adiwinata
author_facet Anurogo, Dito
Yuli Prasetyo Budi, Nova
Thi Ngo, Mai-Huong
Huang, Yen-Hua
Pawitan, Jeanne Adiwinata
author_sort Anurogo, Dito
collection PubMed
description 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.
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spelling pubmed-82307022021-06-26 Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing Anurogo, Dito Yuli Prasetyo Budi, Nova Thi Ngo, Mai-Huong Huang, Yen-Hua Pawitan, Jeanne Adiwinata Int J Mol Sci Review 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. MDPI 2021-06-10 /pmc/articles/PMC8230702/ /pubmed/34200975 http://dx.doi.org/10.3390/ijms22126275 Text en © 2021 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Anurogo, Dito
Yuli Prasetyo Budi, Nova
Thi Ngo, Mai-Huong
Huang, Yen-Hua
Pawitan, Jeanne Adiwinata
Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing
title Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing
title_full Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing
title_fullStr Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing
title_full_unstemmed Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing
title_short Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing
title_sort cell and gene therapy for anemia: hematopoietic stem cells and gene editing
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8230702/
https://www.ncbi.nlm.nih.gov/pubmed/34200975
http://dx.doi.org/10.3390/ijms22126275
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