Gene Therapy for Homozygous β -thalassemia. Is it a Reality?
Author:
Publisher
Informa UK Limited
Subject
Biochemistry (medical),Clinical Biochemistry,Genetics (clinical),Hematology
Link
http://www.tandfonline.com/doi/pdf/10.3109/03630260903351866
Reference25 articles.
1. Phenotype—genotype relationships in monogenic disease: lessons from the thalassaemias
2. Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
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1. Improved Non-Invasive Preimplantation Genetic Testing for Beta-Thalassemia Using Spent Embryo Culture Medium Containing Blastocoelic Fluid;Frontiers in Endocrinology;2022-01-20
2. The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice;Scientific Reports;2016-09-01
3. Improved Hematopoietic Differentiation Efficiency of Gene-Corrected Beta-Thalassemia Induced Pluripotent Stem Cells by CRISPR/Cas9 System;Stem Cells and Development;2015-05
4. Recent trends for novel options in experimental biological therapy of β-thalassemia;Expert Opinion on Biological Therapy;2014-06-16
5. Combining gene therapy and fetal hemoglobin induction for treatment of β-thalassemia;Expert Review of Hematology;2013-06
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