4.8 Article

Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

Related references

Note: Only part of the references are listed.
Article Multidisciplinary Sciences

The TRACE-Seq method tracks recombination alleles and identifies clonal reconstitution dynamics of gene targeted human hematopoietic stem cells

Rajiv Sharma et al.

Summary: Targeted DNA correction in hematopoietic stem and progenitor cells holds promise for treating genetic blood and immune disorders. The utilization of barcoded AAV6 donor template libraries allows for tracking gene-targeted HSPC clones in vivo, potentially advancing translational and basic research in this field.

NATURE COMMUNICATIONS (2021)

Article Biotechnology & Applied Microbiology

Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking

Samuele Ferrari et al.

NATURE BIOTECHNOLOGY (2020)

Article Multidisciplinary Sciences

Gene correction for SCID-X1 in long-term hematopoietic stem cells

Mara Pavel-Dinu et al.

NATURE COMMUNICATIONS (2019)

Article Medicine, General & Internal

CRISPR-Edited Stem Cells in a Patient with HIV and Acute Lymphocytic Leukemia

Lei Xu et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

Article Medicine, Research & Experimental

In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods

Sowmya Pattabhi et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2019)

Article Multidisciplinary Sciences

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I

Natalia Gomez-Ospina et al.

NATURE COMMUNICATIONS (2019)

Article Biochemical Research Methods

CRISPR/Cas9 genome editing in human hematopoietic stem cells

Rasmus O. Bak et al.

NATURE PROTOCOLS (2018)

Article Medicine, General & Internal

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

A. A. Thompson et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Article Medicine, Research & Experimental

Priming Human Repopulating Hematopoietic Stem and Progenitor Cells for Cas9/sgRNA Gene Targeting

Carsten T. Charlesworth et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2018)

Article Cell Biology

CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors

Rasmus O. Bak et al.

CELL REPORTS (2017)

Review Biotechnology & Applied Microbiology

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene

Olivier Negre et al.

HUMAN GENE THERAPY (2016)

Article Multidisciplinary Sciences

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

Daniel P. Dever et al.

NATURE (2016)

Article Cell Biology

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells

Mark A. DeWitt et al.

SCIENCE TRANSLATIONAL MEDICINE (2016)

Review Hematology

α-Globin as a molecular target in the treatment of β-thalassemia

Sachith Mettananda et al.

BLOOD (2015)

Article Multidisciplinary Sciences

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

Matthew C. Canver et al.

NATURE (2015)

Article Biotechnology & Applied Microbiology

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

Ayal Hendel et al.

NATURE BIOTECHNOLOGY (2015)

Article Biochemistry & Molecular Biology

Easy quantitative assessment of genome editing by sequence trace decomposition

Eva K. Brinkman et al.

NUCLEIC ACIDS RESEARCH (2014)

Article Medicine, Research & Experimental

COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites

Thomas J. Cradick et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2014)

Article Medicine, Research & Experimental

The Molecular Basis of β-Thalassemia

Swee Lay Thein

COLD SPRING HARBOR PERSPECTIVES IN MEDICINE (2013)

Article Biotechnology & Applied Microbiology

Universal Real-Time PCR for the Detection and Quantification of Adeno-Associated Virus Serotype 2-Derived Inverted Terminal Repeat Sequences

Christine Aurnhammer et al.

HUMAN GENE THERAPY METHODS (2012)

Article Biochemical Research Methods

FLASH: fast length adjustment of short reads to improve genome assemblies

Tanja Magoc et al.

BIOINFORMATICS (2011)

Article Biochemistry & Molecular Biology

Rapid separation of human globin chains in normal and thalassemia patients by RP-HPLC

Hooshang Nemati et al.

MOLECULAR BIOLOGY REPORTS (2011)

Article Biochemical Research Methods

AAV-mediated gene targeting methods for human cells

Iram F. Khan et al.

NATURE PROTOCOLS (2011)

Article Genetics & Heredity

The impact of single gene and chromosomal disorders on hospital admissions in an adult population

Danielle E. Dye et al.

JOURNAL OF COMMUNITY GENETICS (2011)

Article Multidisciplinary Sciences

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Marina Cavazzana-Calvo et al.

NATURE (2010)

Review Genetics & Heredity

Beta-thalassemia

Renzo Galanello et al.

ORPHANET JOURNAL OF RARE DISEASES (2010)

Article Medicine, General & Internal

Prospects for research in hematologic disorders - Sickle cell disease and thalassemia

WC Mentzer et al.

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2001)