Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

(2021) Chang Li et al.   Blood Advances

Variability in Genome Editing Outcomes: Challenges for Research Reproducibility and Clinical Safety

(2020) Lydia Teboul et al.   MOLECULAR THERAPY

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside

(2020) Olivier Humbert et al.   BRITISH JOURNAL OF HAEMATOLOGY

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

A natural regulatory mutation in the proximal promoter elevates fetal globin expression by creating a de novo GATA1 site

(2019) Gabriella E. Martyn et al.   BLOOD

TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

(2019) Christopher T. Lux et al.   Molecular Therapy-Methods & Clinical Development

Highly efficient therapeutic gene editing of human hematopoietic stem cells

(2019) Yuxuan Wu et al.   NATURE MEDICINE

Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease

(2019) Barbara Cappelli et al.   HAEMATOLOGICA

The clinical application of gene editing: ethical and social issues

(2019) Kelly E Ormond et al.   Personalized Medicine

Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates

(2019) Olivier Humbert et al.   Science Translational Medicine

Health related quality of life among children with transfusion dependent β-thalassaemia major and haemoglobin E β-thalassaemia in Sri Lanka: a case control study

(2019) Sachith Mettananda et al.   Health and Quality of Life Outcomes

New Deletion at Promoter of HBG1 Gene in Sickle Cell Disease Patients With High HbF Level

(2019) Leila Chaouch et al.   JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY

A Survey of Validation Strategies for CRISPR-Cas9 Editing

(2018) Monica F. Sentmanat et al.   Scientific Reports

A Nonhuman Primate Transplantation Model to Evaluate Hematopoietic Stem Cell Gene Editing Strategies for β-Hemoglobinopathies

(2018) Olivier Humbert et al.   Molecular Therapy-Methods & Clinical Development

HLA-E-expressing pluripotent stem cells escape allogeneic responses and lysis by NK cells

(2017) Germán G Gornalusse et al.   NATURE BIOTECHNOLOGY

Sickle Cell Disease

(2017) Frédéric B. Piel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34 + Hematopoietic Stem and Progenitor Cells

(2017) Kai-Hsin Chang et al.   Molecular Therapy-Methods & Clinical Development

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

(2016) Eliane Gluckman et al.   BLOOD

Allogeneic stem cell transplantation for sickle cell disease

(2016) Tara M. Robinson et al.   CURRENT OPINION IN HEMATOLOGY

Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype

(2016) Christian Brendel et al.   JOURNAL OF CLINICAL INVESTIGATION

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

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

Multiplex Genome-Edited T-cell Manufacturing Platform for “Off-the-Shelf” Adoptive T-cell Immunotherapies

(2015) Laurent Poirot et al.   CANCER RESEARCH

Hemoglobin switching's surprise: the versatile transcription factor BCL11A is a master repressor of fetal hemoglobin

(2015) Daniel E Bauer et al.   CURRENT OPINION IN GENETICS & DEVELOPMENT

A Multidrug-resistant Engineered CAR T Cell for Allogeneic Combination Immunotherapy

(2015) Julien Valton et al.   MOLECULAR THERAPY

Genome Editing Technologies: Defining a Path to Clinic

(2015) Jacqueline Corrigan-Curay et al.   MOLECULAR THERAPY

Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel

(2014) E. Angelucci et al.   HAEMATOLOGICA

Fetal hemoglobin in sickle cell anemia: a glass half full?

(2013) M. H. Steinberg et al.   BLOOD

Cancer translocations in human cells induced by zinc finger and TALE nucleases

(2013) M. Piganeau et al.   GENOME RESEARCH

Race/ethnicity affects the probability of finding an HLA-A, -B, -C and -DRB1 allele-matched unrelated donor and likelihood of subsequent transplant utilization

(2012) J Pidala et al.   BONE MARROW TRANSPLANTATION

Discovering the genetics underlying foetal haemoglobin production in adults

(2009) Swee Lay Thein et al.   BRITISH JOURNAL OF HAEMATOLOGY

Global epidemiology of haemoglobin disorders and derived service indicators

(2008) Bernadette Modell   BULLETIN OF THE WORLD HEALTH ORGANIZATION