A novel therapeutic strategy for β-thalassemia (original) (raw)
Efficient gene correction of an aberrant splice site in β‐thalassaemia iPSCs by CRISPR/Cas9 and single‐strand oligodeoxynucleotides
shouheng lin
Journal of Cellular and Molecular Medicine
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Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia
Kevin Luk
Blood, 2019
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Seamless gene correction of beta-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac
Marcus O Muench
Genome Research, 2014
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Correction of Hemoglobin E/Beta-Thalassemia Patient-Derived iPSCs Using CRISPR/Cas9
Methichit Wattanapanitch
Methods in molecular biology, 2021
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Identification of on-target mutagenesis during correction of a beta-thalassemia splice mutation in iPS cells with optimised CRISPR/Cas9-double nickase reveals potential safety concerns
Ernst Wolvetang
APL Bioengineering
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Generation of an in vitro model of β‐thalassemia using the CRISPR/Cas9 genome editing system
Saeid Kaviani
Journal of Cellular Biochemistry, 2019
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Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
Sachith Mettananda
Nature communications, 2017
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One-step genetic correction of hemoglobin E/beta-thalassemia patient-derived iPSCs by the CRISPR/Cas9 system
Nuttha Klincumhom
Stem Cell Research & Therapy
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Universal Gene Correction Approaches for β-hemoglobinopathies Using CRISPR-Cas9 and Adeno-Associated Virus Serotype 6 Donor Templates
Praveen Baskaran
The CRISPR Journal, 2021
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Gene Therapy for B-thalassemia; New Challenges
International Journal of Science and Healthcare Research (IJSHR)
https://ijshr.com/IJSHR\_Vol.5\_Issue.3\_July2020/IJSHR\_Abstract.0025.html, 2020
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A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease
armaan qasba
STEM CELLS Translational Medicine
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Co-Treatment of Erythroid Cells from β-Thalassemia Patients with CRISPR-Cas9-Based β039-Globin Gene Editing and Induction of Fetal Hemoglobin
Lucia Cosenza
Genes
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Genetic correction of haemoglobin E in an immortalised haemoglobin E/beta-thalassaemia cell line using the CRISPR/Cas9 system
Methichit Wattanapanitch
Scientific Reports
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CRISPR, ZFNS, and TALENS for Hemoglobinopathies an Analysis
IJRASET Publication
IJRASET, 2021
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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
Tony W Ho
New England Journal of Medicine, 2021
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Highly efficient therapeutic gene editing of human hematopoietic stem cells
Kevin Luk
Nature Medicine, 2019
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The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements
Michael Antoniou
Journal of Clinical Medicine
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Gene correction of HBB mutations in CD34+ hematopoietic stem cells using Cas9 mRNA and ssODN donors
Praveen Baskaran
Molecular and Cellular Pediatrics, 2018
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Advances in genome editing: the technology of choice for precise and efficient β-thalassemia treatment
Javed Akram
Gene Therapy, 2020
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In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia
Evangelia Yannaki
Journal of Clinical Investigation
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Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements
Zsuzsanna Izsvák
JCI Insight
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Repair of a Splicing Defect in Erythroid Cells from Patients with β-Thalassemia/HbE Disorder
Ryszard Kole
Molecular Therapy, 2002
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Engineered U7 snRNA mediates sustained splicing correction in erythroid cells from β-thalassemia/HbE patients
Suthat Fucharoen
Biochemical and Biophysical Research Communications, 2018
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Recent trends in the gene therapy of β-thalassemia
Nicoletta Bianchi
Journal of Blood Medicine, 2015
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CRISPR to fix bad blood: a new tool in basic and clinical hematology
Rafael Vazquez-Manrique
Haematologica
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Towards More Successful Gene Therapy Clinical Trials for β-Thalassemia
N. Anagnou
Current Molecular Medicine, 2013
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Correction of the Sickle Cell Mutation Through Base and Prime Editing in Hematopoietic Stem Cells
Kelly Ji
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A Review: CRISPR/Cas9-Mediated Genome Editing in Hematopoietic Stem and Progenitor Cells (HSPCs) and Their Applications on Hematological Disorders
LAKSHMI KUMARI
2020
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Hemoglobinopathy Correction with CRISPR or Not; Gene Therapy is the Solution
Bader Al Alwan
Journal of Nature and Science of Medicine, 2020
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Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements
Cecile Masson
Molecular Therapy
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Correction of sickle-cell anemia using CRISPR-Cas9 system in hematopoietic stem cells
Beatriz Draiblate
2020
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High efficiency gene correction in hematopoietic cells by donor-template-free CRISPR/Cas9 genome editing
Stefan Stein
Experimental Hematology, 2017
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Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates
Megan Hoban
Molecular Therapy, 2019
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CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells
Carmen F Bjurström
Molecular Therapy, 2016
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