Related references
Note: Only part of the references are listed.CRISPR technologies for precise epigenome editing
Muneaki Nakamura et al.
NATURE CELL BIOLOGY (2021)
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease
Erica B. Esrick et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
H. Frangoul et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
Transcription factor competition at the γ-globin promoters controls hemoglobin switching
Nan Liu et al.
NATURE GENETICS (2021)
Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice
Adam C. Wilkinson et al.
NATURE COMMUNICATIONS (2021)
Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease
Naoya Uchida et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2021)
Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells
Giulia Pavani et al.
BLOOD ADVANCES (2021)
Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies
Laura Breda et al.
MOLECULAR THERAPY (2021)
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype
Leslie Weber et al.
SCIENCE ADVANCES (2020)
Directed evolution of adenine base editors with increased activity and therapeutic application
Nicole M. Gaudelli et al.
NATURE BIOTECHNOLOGY (2020)
Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity
Michelle F. Richter et al.
NATURE BIOTECHNOLOGY (2020)
Increasing CRISPR Efficiency and Measuring Its Specificity in HSPCs Using a Clinically Relevant System
Jenny Shapiro et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
Combined lentiviral- and RNA-mediated CRISPR/Cas9 delivery for efficient and traceable gene editing in human hematopoietic stem and progenitor cells
David Yudovich et al.
SCIENTIFIC REPORTS (2020)
Evaluation of Homology-Independent CRISPR-Cas9 Off-Target Assessment Methods
Hemangi G. Chaudhari et al.
CRISPR JOURNAL (2020)
Safety and Efficacy of CTX001 in Patients with Transfusion-Dependent β-Thalassemia and Sickle Cell Disease: Early Results from the Climb THAL-111 and Climb SCD-121 Studies of Autologous CRISPR-CAS9-Modified CD34+Hematopoietic Stem and Progenitor Cells
Haydar Frangoul et al.
BLOOD (2020)
Studying DNA Double-Strand Break Repair: An Ever-Growing Toolbox
Alexandra C. Vitor et al.
FRONTIERS IN MOLECULAR BIOSCIENCES (2020)
H4K20meO recognition by BRCA1-BARD1 directs homologous recombination to sister chromatids
Kyosuke Nakamura et al.
NATURE CELL BIOLOGY (2019)
Highly efficient therapeutic gene editing of human hematopoietic stem cells
Yuxuan Wu et al.
NATURE MEDICINE (2019)
TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction
Christopher T. Lux et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2019)
Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease
So Hyun Park et al.
NUCLEIC ACIDS RESEARCH (2019)
Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies
Elisa Magrin et al.
BLOOD (2019)
BCL11A: a potential diagnostic biomarker and therapeutic target in human diseases
Jiawei Yin et al.
BIOSCIENCE REPORTS (2019)
Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia
Angela R. Smith et al.
BLOOD (2019)
Search-and-replace genome editing without double-strand breaks or donor DNA
Andrew V. Anzalone et al.
NATURE (2019)
Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements
Annalisa Lattanzi et al.
MOLECULAR THERAPY (2019)
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent β-thalassemia
Sarah Marktel et al.
NATURE MEDICINE (2019)
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus
Chiara Antoniani et al.
BLOOD (2018)
Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing
Chang Li et al.
BLOOD (2018)
Global Transcriptional Response to CRISPR/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells
M. Kyle Cromer et al.
MOLECULAR THERAPY (2018)
Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding
Gabriella E. Martyn et al.
NATURE GENETICS (2018)
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
A. A. Thompson et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
Leslie Weber et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)
A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells
Marianne Delville et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)
Guide Swap enables genome-scale pooled CRISPR-Cas9 screening in human primary cells
Pamela Y. Ting et al.
NATURE METHODS (2018)
Base editing: precision chemistry on the genome and transcriptome of living cells
Holly A. Rees et al.
NATURE REVIEWS GENETICS (2018)
Gene Therapy for β-Hemoglobinopathies
Marina Cavazzana et al.
MOLECULAR THERAPY (2017)
The control of DNA repair by the cell cycle
Nicole Hustedt et al.
NATURE CELL BIOLOGY (2017)
Non-homologous DNA end joining and alternative pathways to double-strand break repair
Howard H. Y. Chang et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2017)
Gene Therapy in a Patient with Sickle Cell Disease
Jean-Antoine Ribeil et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System
Annalisa Lattanzi et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2017)
Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy
Erika Zonari et al.
STEM CELL REPORTS (2017)
Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing inBone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells
Kai-Hsin Chang et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2017)
Increased risk of leukemia among sickle cell disease patients in California
Ann Brunson et al.
BLOOD (2017)
On Modeling Human Leukocyte Antigen-Identical Sibling Match Probability for Allogeneic Hematopoietic Cell Transplantation: Estimating the Need for an Unrelated Donor Source
Kelsey Besse et al.
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2016)
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype
Christian Brendel et al.
JOURNAL OF CLINICAL INVESTIGATION (2016)
Risk of individual malignant neoplasms in patients with sickle cell disease: English national record linkage study
Olena O. Seminog et al.
JOURNAL OF THE ROYAL SOCIETY OF MEDICINE (2016)
CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
Daniel P. Dever et al.
NATURE (2016)
A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition
Elizabeth A. Traxler et al.
NATURE MEDICINE (2016)
Defining and improving the genome-wide specificities of CRISPR-Cas9 nucleases
Shengdar Q. Tsai et al.
NATURE REVIEWS GENETICS (2016)
Living cell dry mass measurement using quantitative phase imaging with quadriwave lateral shearing interferometry: an accuracy and sensitivity discussion
Sherazade Aknoun et al.
JOURNAL OF BIOMEDICAL OPTICS (2015)
BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis
Matthew C. Canver et al.
NATURE (2015)
CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes
Puping Liang et al.
PROTEIN & CELL (2015)
Integrative Analysis of CRISPR/Cas9 Target Sites in the Human HBB Gene
Yumei Luo et al.
BIOMED RESEARCH INTERNATIONAL (2015)
Optimizing sgRNA structure to improve CRISPR-Cas9 knockout efficiency
Ying Dang et al.
GENOME BIOLOGY (2015)
Fetal hemoglobin in sickle cell anemia: a glass half full?
Martin H. Steinberg et al.
BLOOD (2014)
Targeted genome editing in human repopulating haematopoietic stem cells
Pietro Genovese et al.
NATURE (2014)
Easy quantitative assessment of genome editing by sequence trace decomposition
Eva K. Brinkman et al.
NUCLEIC ACIDS RESEARCH (2014)
COSMID: A Web-based Tool for Identifying and Validating CRISPR/Cas Off-target Sites
Thomas J. Cradick et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2014)
Causes of Death in Sickle Cell Disease Adult Patients: Old and New Trends
Stephanie Ngo et al.
BLOOD (2014)
CRISPR/Cas9 systems targeting β-globin and CCR5 genes have substantial off-target activity
Thomas J. Cradick et al.
NUCLEIC ACIDS RESEARCH (2013)
Establishment of Immortalized Human Erythroid Progenitor Cell Lines Able to Produce Enucleated Red Blood Cells
Ryo Kurita et al.
PLOS ONE (2013)
The GATA1-HS2 Enhancer Allows Persistent and Position-Independent Expression of a β-globin Transgene
Annarita Miccio et al.
PLOS ONE (2011)
Correction of β-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients
Emanuela Anna Roselli et al.
EMBO MOLECULAR MEDICINE (2010)
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Marina Cavazzana-Calvo et al.
NATURE (2010)
Beta-thalassemia
Renzo Galanello et al.
ORPHANET JOURNAL OF RARE DISEASES (2010)
Quadriwave lateral shearing interferometry for quantitative phase microscopy of living cells
Pierre Bon et al.
OPTICS EXPRESS (2009)
In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of β-thalassemia
Annarita Miccio et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)
Human Fetal Hemoglobin Expression Is Regulated by the Developmental Stage-Specific Repressor BCL11A
Vijay G. Sankaran et al.
SCIENCE (2008)
Therapeutic options for patients with severe beta-thalassemia: The need for globin gene therapy
Michel Sadelain et al.
HUMAN GENE THERAPY (2007)
Ex vivo generation of fully mature human red blood cells from hematopoietic stem cells
MC Giarratana et al.
NATURE BIOTECHNOLOGY (2005)
A recombinant human hemoglobin with anti-sickling properties greater than fetal hemoglobin
DN Levasseur et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2004)
Stern cell transplantation for hemoglobinopathies
J Gaziev et al.
CURRENT OPINION IN PEDIATRICS (2003)
Share Paper
