Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells

Tandem Paired Nicking Promotes Precise Genome Editing with Scarce Interference by p53

(2020) Toshinori Hyodo et al.   Cell Reports

Partner independent fusion gene detection by multiplexed CRISPR-Cas9 enrichment and long read nanopore sequencing

(2020) Christina Stangl et al.   Nature Communications

Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins

(2020) Giulia Pavani et al.   Nature Communications

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

(2019) Sarah Marktel et al.   NATURE MEDICINE

CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations

(2019) Grégoire Cullot et al.   Nature Communications

Highly efficient therapeutic gene editing of human hematopoietic stem cells

(2019) Yuxuan Wu et al.   NATURE MEDICINE

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

CRISPResso2 provides accurate and rapid genome editing sequence analysis

(2019) Kendell Clement et al.   NATURE BIOTECHNOLOGY

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

(2019) Giulia Schiroli et al.   Cell Stem Cell

Introducing a Spectrum of Long-Range Genomic Deletions in Human Embryonic Stem Cells Using Type I CRISPR-Cas

(2019) Adam E. Dolan et al.   MOLECULAR 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

Alpha Globin Gene Mutation: A Major Determinant of Hydroxyurea Response in Transfusion-Dependent HbE-β-Thalassaemia

(2019) Sujana Biswas et al.   ACTA HAEMATOLOGICA

Development of hRad51–Cas9 nickase fusions that mediate HDR without double-stranded breaks

(2019) Holly A. Rees et al.   Nature Communications

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

(2019) Mara Pavel-Dinu et al.   Nature Communications

High levels of AAV vector integration into CRISPR-induced DNA breaks

(2019) Killian S. Hanlon et al.   Nature Communications

Synergistic silencing of α-globin and induction of γ-globin by histone deacetylase inhibitor, vorinostat as a potential therapy for β-thalassaemia

(2019) Sachith Mettananda et al.   Scientific Reports

Polymer-stabilized Cas9 nanoparticles and modified repair templates increase genome editing efficiency

(2019) David N. Nguyen et al.   NATURE BIOTECHNOLOGY

Expanding the editable genome and CRISPR–Cas9 versatility using DNA cutting-free gene targeting based on in trans paired nicking

(2019) Xiaoyu Chen et al.   NUCLEIC ACIDS RESEARCH

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

(2018) Chiara Antoniani et al.   BLOOD

Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing

(2018) Chang Li et al.   BLOOD

Variability of homozygous sickle cell disease: The role of alpha and beta globin chain variation and other factors

(2018) Graham R. Serjeant et al.   BLOOD CELLS MOLECULES AND DISEASES

Molecular Basis and Genetic Modifiers of Thalassemia

(2018) Sachith Mettananda et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding

(2018) Gabriella E. Martyn et al.   NATURE GENETICS

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

(2018) Alexis A. Thompson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Common α-globin variants modify hematologic and other clinical phenotypes in sickle cell trait and disease

(2018) Laura M. Raffield et al.   PLoS Genetics

Large deletions induced by Cas9 cleavage

(2018) Fatwa Adikusuma et al.   NATURE

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

(2018) Leslie Weber et al.   Molecular Therapy-Methods & Clinical Development

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

(2018) Annalisa Lattanzi et al.   MOLECULAR THERAPY

Cellular function reinstitution of offspring red blood cells cloned from the sickle cell disease patient blood post CRISPR genome editing

(2017) Jianguo Wen et al.   Journal of Hematology & Oncology

Management of iron overload in hemoglobinopathies

(2017) S. Allali et al.   TRANSFUSION CLINIQUE ET BIOLOGIQUE

Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia

(2017) Sachith Mettananda et al.   Nature Communications

In trans paired nicking triggers seamless genome editing without double-stranded DNA cutting

(2017) Xiaoyu Chen et al.   Nature Communications

Next-generation mapping: a novel approach for detection of pathogenic structural variants with a potential utility in clinical diagnosis

(2017) Hayk Barseghyan et al.   Genome Medicine

On Modeling Human Leukocyte Antigen–Identical Sibling Match Probability for Allogeneic Hematopoietic Cell Transplantation: Estimating the Need for an Unrelated Donor Source

(2016) Kelsey Besse et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Selective silencing of α-globin by the histone demethylase inhibitor IOX1: a potentially new pathway for treatment of β-thalassemia

(2016) Sachith Mettananda et al.   HAEMATOLOGICA

Plerixafor and G-CSF combination mobilizes hematopoietic stem and progenitors cells with a distinct transcriptional profile and a reduced in vivo homing capacity compared to plerixafor alone

(2016) Maria Rosa Lidonnici et al.   HAEMATOLOGICA

Open-source guideseq software for analysis of GUIDE-seq data

(2016) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition

(2016) Elizabeth A Traxler et al.   NATURE MEDICINE

Selective silencing of α-globin by the histone demethylase inhibitor IOX1: a potentially new pathway for treatment of β-thalassemia

(2016) Sachith Mettananda et al.   HAEMATOLOGICA

Plerixafor and G-CSF combination mobilizes hematopoietic stem and progenitors cells with a distinct transcriptional profile and a reduced in vivo homing capacity compared to plerixafor alone

(2016) Maria Rosa Lidonnici et al.   HAEMATOLOGICA

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

(2015) S. Mettananda et al.   BLOOD

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

(2015) Jianbin Wang et al.   NATURE BIOTECHNOLOGY

Efficient inversions and duplications of mammalian regulatory DNA elements and gene clusters by CRISPR/Cas9

(2015) Jinhuan Li et al.   Journal of Molecular Cell Biology

Homology-directed repair in rodent zygotes using Cas9 and TALEN engineered proteins

(2015) Séverine Ménoret et al.   Scientific Reports

Biosafety Challenges for Use of Lentiviral Vectors in Gene Therapy

(2014) Michael Rothe et al.   CURRENT GENE THERAPY

Gene Therapy for Hemoglobinopathies

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

The Correlation of α-Globin Gene Mutations and theXmnI Polymorphism with Clinical Severity of Hb E/β-Thalassemia

(2014) Pimlak Charoenkwan et al.   HEMOGLOBIN

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

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

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Easy quantitative assessment of genome editing by sequence trace decomposition

(2014) Eva K. Brinkman et al.   NUCLEIC ACIDS RESEARCH

The Co-Inheritance of Alpha-Thalassemia and Sickle Cell Anemia Is Associated with Better Hematological Indices and Lower Consultations Rate in Cameroonian Patients and Could Improve Their Survival

(2014) Maryam Bibi Rumaney et al.   PLoS One

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

PARP-mediated repair, homologous recombination, and back-up non-homologous end joining-like repair of single-strand nicks

(2013) Michael J. Metzger et al.   DNA REPAIR

A Double-Switch Vector System Positively Regulates Transgene Expression by Endogenous microRNA Expression (miR-ON Vector)

(2013) Mario Amendola et al.   MOLECULAR THERAPY

Establishment of Immortalized Human Erythroid Progenitor Cell Lines Able to Produce Enucleated Red Blood Cells

(2013) Ryo Kurita et al.   PLoS One

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

The Hemoglobin E Thalassemias

(2013) S. Fucharoen et al.   Cold Spring Harbor Perspectives in Medicine

The Molecular Basis of  -Thalassemia

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

Outcomes after matched unrelated donor versus identical sibling hematopoietic cell transplantation in adults with acute myelogenous leukemia

(2012) W. Saber et al.   BLOOD

The emerging role of fetal hemoglobin induction in non-transfusion-dependent thalassemia

(2012) Swee Lay Thein   BLOOD REVIEWS

Macrophages prevent human red blood cell reconstitution in immunodeficient mice

(2011) Z. Hu et al.   BLOOD

The challenge of haemoglobinopathies in resource-poor countries

(2011) David J. Weatherall   BRITISH JOURNAL OF HAEMATOLOGY

Comparative analysis of FV vectors with human α- or β-globin gene regulatory elements for the correction of β-thalassemia

(2011) I Morianos et al.   GENE THERAPY

Lethal toxicity caused by expression of shRNA in the mouse striatum: implications for therapeutic design

(2011) J N Martin et al.   GENE THERAPY

Variants in genetic modifiers of  -thalassemia can help to predict the major or intermedia type of the disease

(2011) C. Badens et al.   HAEMATOLOGICA

The New Self-Inactivating Lentiviral Vector for Thalassemia Gene Therapy Combining Two HPFH Activating Elements Corrects Human Thalassemic Hematopoietic Stem Cells

(2011) Eleni Papanikolaou et al.   HUMAN GENE THERAPY

Correction of β654-thalassaemia mice using direct intravenous injection of siRNA and antisense RNA vectors

(2011) Shu-Yang Xie et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY

Production, Purification and Characterization of Adeno-Associated Vectors

(2010) Eduard Ayuso et al.   CURRENT GENE THERAPY

Beta-thalassemia

(2010) Antonio Cao et al.   GENETICS IN MEDICINE

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

(2010) Marina Cavazzana-Calvo et al.   NATURE

Single-strand nicks induce homologous recombination with less toxicity than double-strand breaks using an AAV vector template

(2010) Michael J. Metzger et al.   NUCLEIC ACIDS RESEARCH

α-thalassaemia

(2010) Cornelis L Harteveld et al.   Orphanet Journal of Rare Diseases

Lentiviral delivery of short hairpin RNAs

(2009) N. Manjunath et al.   ADVANCED DRUG DELIVERY REVIEWS

Effect of α-gene numbers on phenotype of HbE/β thalassemia patients

(2009) Vineeta Sharma et al.   ANNALS OF HEMATOLOGY

siRNA-mediated reduction of  -globin results in phenotypic improvements in  -thalassemic cells

(2008) H. P. J. Voon et al.   HAEMATOLOGICA

Single-strand break repair and genetic disease

(2008) Keith W. Caldecott   NATURE REVIEWS GENETICS

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of  -thalassemia

(2008) A. Miccio et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA