Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice

In vivo HSC prime editing rescues Sickle Cell Disease in a mouse model

(2023) Chang Li et al.   BLOOD

Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins

(2022) Samagya Banskota et al.   CELL

Prime editing efficiency and fidelity are enhanced in the absence of mismatch repair

(2022) J. Ferreira da Silva et al.   Nature Communications

Genomic and Transcriptomic Analyses of Prime Editing Guide RNA–Independent Off-Target Effects by Prime Editors

(2022) Runze Gao et al.   CRISPR Journal

High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation

(2022) Wendy Magis et al.   iScience

Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells

(2022) Samuele Ferrari et al.   Cell Stem Cell

Prime editing for precise and highly versatile genome manipulation

(2022) Peter J. Chen et al.   NATURE REVIEWS GENETICS

Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

(2021) Adam C. Wilkinson et al.   Nature Communications

High-efficiency prime editing with optimized, paired pegRNAs in plants

(2021) Qiupeng Lin et al.   NATURE BIOTECHNOLOGY

Genome-wide specificity of prime editors in plants

(2021) Shuai Jin et al.   NATURE BIOTECHNOLOGY

Rationally Designed Base Editors for Precise Editing of the Sickle Cell Disease Mutation

(2021) S. Haihua Chu et al.   CRISPR Journal

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

(2021) Gregory A. Newby et al.   NATURE

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

(2021) Annalisa Lattanzi et al.   Science Translational Medicine

Modeling a cataract disorder in mice with prime editing

(2021) Jianxiang Lin et al.   Molecular Therapy-Nucleic Acids

Evaluating CRISPR-based prime editing for cancer modeling and CFTR repair in organoids

(2021) Maarten H Geurts et al.   Life Science Alliance

Enhanced prime editing systems by manipulating cellular determinants of editing outcomes

(2021) Peter J. Chen et al.   CELL

Engineered pegRNAs improve prime editing efficiency

(2021) James W. Nelson et al.   NATURE BIOTECHNOLOGY

Precise genomic deletions using paired prime editing

(2021) Junhong Choi et al.   NATURE BIOTECHNOLOGY

Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing

(2021) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease

(2021) Julie Kanter et al.   NEW ENGLAND JOURNAL OF MEDICINE

Comprehensive analysis of prime editing outcomes in human embryonic stem cells

(2021) Omer Habib et al.   NUCLEIC ACIDS RESEARCH

Intracellular RNase activity dampens zinc finger nuclease-mediated gene editing in hematopoietic stem and progenitor cells

(2021) Christopher W. Peterson et al.   Molecular Therapy-Methods & Clinical Development

Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

(2020) Samuel Lessard et al.   BLOOD

Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors

(2020) Jordan L. Doman et al.   NATURE BIOTECHNOLOGY

Cas9 activates the p53 pathway and selects for p53-inactivating mutations

(2020) Oana M. Enache et al.   NATURE GENETICS

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

Efficient generation of mouse models with the prime editing system

(2020) Yao Liu et al.   Cell Discovery

p53 Hinders CRISPR/Cas9-Mediated Targeted Gene Disruption in Memory CD8 T Cells In Vivo

(2020) Samarchith P. Kurup et al.   JOURNAL OF IMMUNOLOGY

Unbiased investigation of specificities of prime editing systems in human cells

(2020) Do Yon Kim et al.   NUCLEIC ACIDS RESEARCH

Prime editing for functional repair in patient-derived disease models

(2020) Imre F. Schene et al.   Nature Communications

Allele-Specific Chromosome Removal after Cas9 Cleavage in Human Embryos

(2020) Michael V. Zuccaro et al.   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

Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos

(2019) Erwei Zuo et al.   SCIENCE

Cytosine, but not adenine, base editors induce genome-wide off-target mutations in rice

(2019) Shuai Jin et al.   SCIENCE

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

(2019) Giulia Schiroli 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

In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods

(2019) Sowmya Pattabhi et al.   Molecular Therapy-Nucleic Acids

EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD

(2019) Edouard De Dreuzy et al.   BLOOD

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

Low-Dose Busulfan Reduces Human CD34+ Cell Doses Required for Engraftment in c-kit Mutant Immunodeficient Mice

(2019) Alexis Leonard et al.   Molecular Therapy-Methods & Clinical Development

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

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells

(2018) Robert J. Ihry et al.   NATURE MEDICINE

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

(2018) Emma Haapaniemi et al.   NATURE MEDICINE

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR–Cas9 nuclease off-targets

(2017) Shengdar Q Tsai et al.   NATURE METHODS

Sickle Cell Disease

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

Advances in the delivery of RNA therapeutics: from concept to clinical reality

(2017) James C. Kaczmarek et al.   Genome Medicine

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

(2016) Daniel P. Dever 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

Bcl11a Deficiency Leads to Hematopoietic Stem Cell Defects with an Aging-like Phenotype

(2016) Sidinh Luc et al.   Cell Reports

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

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Nonirradiated NOD,B6.SCID Il2rγ−/− KitW41/W41 (NBSGW) Mice Support Multilineage Engraftment of Human Hematopoietic Cells

(2015) Brian E. McIntosh et al.   Stem Cell Reports

Isolation and functional characterization of human erythroblasts at distinct stages: implications for understanding of normal and disordered erythropoiesis in vivo

(2013) J. Hu et al.   BLOOD

Ineffective Erythropoiesis inβ-Thalassemia

(2013) Jean-Antoine Ribeil et al.   TheScientificWorldJOURNAL

The Switch from Fetal to Adult Hemoglobin

(2013) V. G. Sankaran et al.   Cold Spring Harbor Perspectives in Medicine

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

(2010) Stefan Stein et al.   NATURE MEDICINE

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION