CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease

Transcription factor competition at the γ-globin promoters controls hemoglobin switching

(2021) Nan Liu et al.   NATURE GENETICS

Persistence of CRISPR/Cas9 gene edited hematopoietic stem cells following transplantation: A systematic review and meta‐analysis of preclinical studies

(2021) Harinad B. Maganti et al.   Stem Cells Translational Medicine

The Cas9 Hammer—and Sickle: A Challenge for Genome Editors

(2021) Fyodor D. Urnov   CRISPR Journal

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

Epigenetic inactivation of ERF reactivates γ-globin expression in β-thalassemia

(2021) Xiuqin Bao et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Genetic therapies for the first molecular disease

(2021) Phillip A. Doerfler et al.   JOURNAL OF CLINICAL INVESTIGATION

A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease

(2021) Alexis Leonard et al.   MOLECULAR THERAPY

Single-nucleotide-level mapping of DNA regulatory elements that control fetal hemoglobin expression

(2021) Li Cheng et al.   NATURE GENETICS

ZNF410 represses fetal globin by singular control of CHD4

(2021) Divya S. Vinjamur et al.   NATURE GENETICS

Molecular analysis of the erythroid phenotype of a patient with BCL11A haploinsufficiency

(2021) Marja W. Wessels et al.   Blood Advances

Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease

(2021) Alexis Leonard et al.   Blood Advances

Reactivation of γ-globin expression through Cas9 or base editor to treat β-hemoglobinopathies

(2020) Liren Wang et al.   CELL RESEARCH

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

(2020) Leslie Weber et al.   Science Advances

HRI-regulated transcription factor ATF4 activates BCL11A transcription to silence fetal hemoglobin expression

(2020) Peng Huang et al.   BLOOD

Genome editing strategies for fetal hemoglobin induction in beta-hemoglobinopathies

(2020) Selami Demirci et al.   HUMAN MOLECULAR GENETICS

Absolute Quantification of Transcription Factors Reveals Principles of Gene Regulation in Erythropoiesis

(2020) Mark A. Gillespie et al.   MOLECULAR CELL

Directed evolution of adenine base editors with increased activity and therapeutic application

(2020) Nicole M. Gaudelli et al.   NATURE BIOTECHNOLOGY

Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity

(2020) Michelle F. Richter et al.   NATURE BIOTECHNOLOGY

Dual base editor catalyzes both cytosine and adenine base conversions in human cells

(2020) Xiaohui Zhang et al.   NATURE BIOTECHNOLOGY

Increasing the efficiency and targeting range of cytidine base editors through fusion of a single-stranded DNA-binding protein domain

(2020) Xiaohui Zhang et al.   NATURE CELL BIOLOGY

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

(2020) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

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

Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease

(2020) Matthew M. Hsieh et al.   Blood Advances

In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model

(2020) Chang Li et al.   MOLECULAR THERAPY

HRI depletion cooperates with pharmacologic inducers to elevate fetal hemoglobin and reduce sickle cell formation

(2020) Scott A. Peslak et al.   Blood Advances

A natural DNMT1 mutation elevates the fetal hemoglobin via epigenetic de-repression of γ-globin gene in β-thalassemia

(2020) Yi Gong et al.   BLOOD

Induction of therapeutic levels of HbF in genome‐edited primary β 0 39‐thalassaemia haematopoietic stem and progenitor cells

(2020) Maura Mingoia et al.   BRITISH JOURNAL OF HAEMATOLOGY

ZNF410 Uniquely Activates the NuRD Component CHD4 to Silence Fetal Hemoglobin Expression

(2020) Xianjiang Lan et al.   MOLECULAR CELL

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

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

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

Hematopoietic chimerism and donor-specific skin allograft tolerance after non-genotoxic CD117 antibody-drug-conjugate conditioning in MHC-mismatched allotransplantation

(2019) Zhanzhuo Li et al.   Nature Communications

Highly efficient therapeutic gene editing of human hematopoietic stem cells

(2019) Yuxuan Wu et al.   NATURE MEDICINE

Disruption of the MBD2-NuRD complex but not MBD3-NuRD induces high level HbF expression in human erythroid cells

(2019) Xiaofei Yu et al.   HAEMATOLOGICA

A novel mutation in the erythroid transcription factor KLF1 is likely responsible for ameliorating β‐thalassemia major

(2019) Pavlos Fanis et al.   HUMAN MUTATION

Rational targeting of a NuRD subcomplex guided by comprehensive in situ mutagenesis

(2019) Falak Sher et al.   NATURE GENETICS

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

A new target for fetal hemoglobin reactivation

(2019) Angela Rivers et al.   HAEMATOLOGICA

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

Direct Promoter Repression by BCL11A Controls the Fetal to Adult Hemoglobin Switch

(2018) Nan Liu et al.   CELL

Gene therapy for sickle cell disease: An update

(2018) Selami Demirci et al.   CYTOTHERAPY

Fetal hemoglobin induction in sickle erythroid progenitors using a synthetic zinc finger DNA-binding domain

(2018) Biaoru Li et al.   HAEMATOLOGICA

Disruption of SOX6 gene using CRISPR/Cas9 technology for gamma-globin reactivation: An approach towards gene therapy of β-thalassemia

(2018) Laleh Shariati et al.   JOURNAL OF CELLULAR BIOCHEMISTRY

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

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction

(2018) Luke W Koblan 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

CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response

(2018) Emma Haapaniemi et al.   NATURE MEDICINE

Domain-focused CRISPR screen identifies HRI as a fetal hemoglobin regulator in human erythroid cells

(2018) Jeremy D. Grevet et al.   SCIENCE

Fetal hemoglobin induction in sickle erythroid progenitors using a synthetic zinc finger DNA-binding domain

(2018) Biaoru Li et al.   HAEMATOLOGICA

Gene therapy: evidence, value and affordability in the US health care system

(2018) Grace Hampson et al.   Journal of Comparative Effectiveness Research

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

Wake-up Sleepy Gene: Reactivating Fetal Globin for β-Hemoglobinopathies

(2018) Beeke Wienert et al.   TRENDS IN GENETICS

CRISPR-Cas orthologues and variants: optimizing the repertoire, specificity and delivery of genome engineering tools

(2017) Alberto Cebrian-Serrano et al.   MAMMALIAN GENOME

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

CRISPR/Cas9 targeting events cause complex deletions and insertions at 17 sites in the mouse genome

(2017) Ha Youn Shin et al.   Nature Communications

BCL11A Haploinsufficiency Causes an Intellectual Disability Syndrome and Dysregulates Transcription

(2016) Cristina Dias et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Beyond hydroxyurea: new and old drugs in the pipeline for sickle cell disease

(2016) M. J. Telen   BLOOD

Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype

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

Genetic disruption of theKLF1gene to overexpress the γ-globin gene using the CRISPR/Cas9system

(2016) Laleh Shariati et al.   JOURNAL OF GENE MEDICINE

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin

(2016) Rahul Palchaudhuri 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

Defining Sickle Cell Disease Mortality Using a Population-Based Surveillance System, 2004 through 2008

(2016) Susan T. Paulukonis et al.   PUBLIC HEALTH REPORTS

Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin

(2016) T. Masuda et al.   SCIENCE

EHMT1 and EHMT2 inhibition induces fetal hemoglobin expression

(2015) A. Renneville et al.   BLOOD

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

BCL11A deletions result in fetal hemoglobin persistence and neurodevelopmental alterations

(2015) Anindita Basak et al.   JOURNAL OF CLINICAL INVESTIGATION

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

Targeted genome editing in human repopulating haematopoietic stem cells

(2014) Pietro Genovese et al.   NATURE

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

(2013) M. H. Steinberg et al.   BLOOD

The NuRD architecture

(2013) Hillary F. Allen et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Transcriptional regulators Myb and BCL11A interplay with DNA methyltransferase 1 in developmental silencing of embryonic and fetal β-like globin genes

(2013) Mark Roosjen et al.   FASEB JOURNAL

Probing the Dynamic Distribution of Bound States for Methylcytosine-binding Domains on DNA

(2013) Jason M. Cramer et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Lysine-specific demethylase 1 is a therapeutic target for fetal hemoglobin induction

(2013) Lihong Shi et al.   NATURE MEDICINE

Corepressor-dependent silencing of fetal hemoglobin expression by BCL11A

(2013) J. Xu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

An Erythroid Enhancer of BCL11A Subject to Genetic Variation Determines Fetal Hemoglobin Level

(2013) D. E. Bauer et al.   SCIENCE

Ldb1 complexes: the new master regulators of erythroid gene transcription

(2013) Paul E. Love et al.   TRENDS IN GENETICS

Reawakening fetal hemoglobin: prospects for new therapies for the  -globin disorders

(2012) D. E. Bauer et al.   BLOOD

MBD2 and Multiple Domains of CHD4 Are Required for Transcriptional Repression by Mi-2/NuRD Complexes

(2012) J. Ramirez et al.   MOLECULAR AND CELLULAR BIOLOGY

Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG)

(2011) Winfred C Wang et al.   LANCET

A Functional Element Necessary for Fetal Hemoglobin Silencing

(2011) Vijay G. Sankaran et al.   NEW ENGLAND JOURNAL OF MEDICINE

Correction of Sickle Cell Disease in Adult Mice by Interference with Fetal Hemoglobin Silencing

(2011) J. Xu et al.   SCIENCE

How I use hydroxyurea to treat young patients with sickle cell anemia

(2010) R. E. Ware   BLOOD

Transcriptional silencing of  -globin by BCL11A involves long-range interactions and cooperation with SOX6

(2010) J. Xu et al.   GENES & DEVELOPMENT

LRF Is an Essential Downstream Target of GATA1 in Erythroid Development and Regulates BIM-Dependent Apoptosis

(2009) Takahiro Maeda et al.   DEVELOPMENTAL CELL

Genome-wide association study shows BCL11A associated with persistent fetal hemoglobin and amelioration of the phenotype of  -thalassemia

(2008) M. Uda et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA