Universal Gene Correction Approaches for β-hemoglobinopathies Using CRISPR-Cas9 and Adeno-Associated Virus Serotype 6 Donor Templates

Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin

(2020) Andrés Lamsfus-Calle et al.   Scientific Reports

Comparative analysis of lentiviral gene transfer approaches designed to promote fetal hemoglobin production for the treatment of β-hemoglobinopathies

(2020) Alberto Daniel-Moreno et al.   BLOOD CELLS MOLECULES AND DISEASES

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

(2020) Erica B. Esrick et al.   NEW ENGLAND JOURNAL OF MEDICINE

Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

(2019) Giulia Schiroli et al.   Cell Stem Cell

Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination

(2019) Renata M. Martin et al.   Cell Stem Cell

Editing the Sickle Cell Disease Mutation in Primary Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates

(2019) Zulema Romero et al.   MOLECULAR THERAPY

CHOPCHOP v3: expanding the CRISPR web toolbox beyond genome editing

(2019) Kornel Labun et al.   NUCLEIC ACIDS RESEARCH

Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease

(2019) So Hyun Park et al.   NUCLEIC ACIDS RESEARCH

Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies

(2019) Elisa Magrin et al.   BLOOD

Hematopoietic stem cell gene therapy: The optimal use of lentivirus and gene editing approaches

(2019) Andrés Lamsfus-Calle et al.   BLOOD REVIEWS

Efficient gene correction of an aberrant splice site in β‐thalassaemia iPSCs by CRISPR/Cas9 and single‐strand oligodeoxynucleotides

(2019) Zeyu Xiong et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

(2018) Christopher A. Vakulskas et al.   NATURE MEDICINE

Adeno-Associated Virus Vectors and Stem Cells: Friends or Foes?

(2017) Nolan Brown et al.   HUMAN GENE THERAPY

A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease

(2017) Liuhong Cai et al.   Stem Cells Translational Medicine

CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells

(2016) Megan D Hoban et al.   MOLECULAR THERAPY

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

(2016) Daniel P. Dever et al.   NATURE

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

(2016) M. A. DeWitt et al.   Science Translational Medicine

Identification and characterization of human nucleus pulposus cell specific serotypes of adeno-associated virus for gene therapeutic approaches of intervertebral disc disorders

(2015) Demissew S. Mern et al.   BMC MUSCULOSKELETAL DISORDERS

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

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Evidence-based focused review of the status of hematopoietic stem cell transplantation as treatment of sickle cell disease and thalassemia

(2014) A. King et al.   BLOOD

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 andpiggyBac

(2014) Fei Xie et al.   GENOME RESEARCH

Gene Therapy for Hemoglobinopathies

(2014) Shanmuganathan Chandrakasan et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

IthaGenes: An Interactive Database for Haemoglobin Variations and Epidemiology

(2014) Petros Kountouris et al.   PLoS One

Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo

(2013) Liujiang Song et al.   CYTOTHERAPY

Pathophysiology and Clinical Manifestations of the  -Thalassemias

(2013) A. W. Nienhuis et al.   Cold Spring Harbor Perspectives in Medicine

Management of the Thalassemias

(2013) N. F. Olivieri et al.   Cold Spring Harbor Perspectives in Medicine

The Molecular Basis of  -Thalassemia

(2013) S. L. Thein   Cold Spring Harbor Perspectives in Medicine

Fetal hemoglobin in sickle cell anemia: Molecular characterization of the unusually high fetal hemoglobin phenotype in African Americans

(2011) Idowu Akinsheye et al.   AMERICAN JOURNAL OF HEMATOLOGY

Thalassaemia

(2011) Douglas R Higgs et al.   LANCET

Beta-thalassemia

(2010) Renzo Galanello et al.   Orphanet Journal of Rare Diseases