Correction of Hemoglobin E/Beta-Thalassemia Patient-Derived iPSCs Using CRISPR/Cas9 (original) (raw)
Genetic correction of haemoglobin E in an immortalised haemoglobin E/beta-thalassaemia cell line using the CRISPR/Cas9 system
Methichit Wattanapanitch
Scientific Reports
View PDFchevron_right
Seamless gene correction of beta-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac
Marcus O Muench
Genome Research, 2014
View PDFchevron_right
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
View PDFchevron_right
One-step genetic correction of hemoglobin E/beta-thalassemia patient-derived iPSCs by the CRISPR/Cas9 system
Methichit Wattanapanitch
Stem Cell Research & Therapy
View PDFchevron_right
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
View PDFchevron_right
Universal Gene Correction Approaches for β-hemoglobinopathies Using CRISPR-Cas9 and Adeno-Associated Virus Serotype 6 Donor Templates
Praveen Baskaran
The CRISPR Journal, 2021
View PDFchevron_right
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
View PDFchevron_right
Generation of an in vitro model of β‐thalassemia using the CRISPR/Cas9 genome editing system
Saeid Kaviani
Journal of Cellular Biochemistry, 2019
View PDFchevron_right
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
View PDFchevron_right
Hemoglobinopathy Correction with CRISPR or Not; Gene Therapy is the Solution
Bader Al Alwan
Journal of Nature and Science of Medicine, 2020
View PDFchevron_right
Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
Sachith Mettananda
Nature communications, 2017
View PDFchevron_right
Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β039-thalassemia patients
Lucia Cosenza
Molecular Therapy - Methods & Clinical Development
View PDFchevron_right
Advances in genome editing: the technology of choice for precise and efficient β-thalassemia treatment
Javed Akram
Gene Therapy, 2020
View PDFchevron_right
Gene correction of HBB mutations in CD34+ hematopoietic stem cells using Cas9 mRNA and ssODN donors
Praveen Baskaran
Molecular and Cellular Pediatrics, 2018
View PDFchevron_right
CRISPR, ZFNS, and TALENS for Hemoglobinopathies an Analysis
IJRASET Publication
IJRASET, 2021
View PDFchevron_right
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
Tony W Ho
New England Journal of Medicine, 2021
View PDFchevron_right
Production of gene-corrected adult beta globin protein in human erythrocytes differentiated from patient iPSCs after genome editing of the sickle point mutation
Cory Smith
Stem cells (Dayton, Ohio), 2015
View PDFchevron_right
Gene Editing-Based Technologies for Beta-hemoglobinopathies Treatment
Elham Bidram
Biology
View PDFchevron_right
Identification of novel HPFH-like mutations by CRISPR base editing that elevates the expression of fetal hemoglobin
Aswin anand pai
2020
View PDFchevron_right
Highly efficient therapeutic gene editing of human hematopoietic stem cells
Kevin Luk
Nature Medicine, 2019
View PDFchevron_right
Towards More Successful Gene Therapy Clinical Trials for β-Thalassemia
N. Anagnou
Current Molecular Medicine, 2013
View PDFchevron_right
Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin
Praveen Baskaran
Scientific Reports, 2020
View PDFchevron_right
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
View PDFchevron_right
Recent trends in the gene therapy of β-thalassemia
Nicoletta Bianchi
Journal of Blood Medicine, 2015
View PDFchevron_right
A novel therapeutic strategy for β-thalassemia
Tim Townes
Blood, 2019
View PDFchevron_right
Targeted deletion of BCL11A gene by CRISPR-Cas9 system for fetal hemoglobin reactivation: A promising approach for gene therapy of beta thalassemia disease
Arash Arashkia
View PDFchevron_right
The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements
Michael Antoniou
Journal of Clinical Medicine
View PDFchevron_right
Correction of sickle-cell anemia using CRISPR-Cas9 system in hematopoietic stem cells
Beatriz Draiblate
2020
View PDFchevron_right
Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin
kirti prasad
eLife
View PDFchevron_right
Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia
Kevin Luk
Blood, 2019
View PDFchevron_right
A Review of CRISPR Cas9 for SCA: Treatment Strategies and Could Target β-globin Gene and BCL11A Gene using CRISPR Cas9 Prevent the Patient from Sickle Cell Anemia?
arga setyo adji
Open Access Macedonian Journal of Medical Sciences
View PDFchevron_right
Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements
Fatima Amor
Molecular Therapy, 2018
View PDFchevron_right