Correction of Beta-Thalassemia IVS-II-654 Mutation in a Mouse Model Using Prime Editing

Engineered prime editors with PAM flexibility

(2021) Jiyeon Kweon et al.   MOLECULAR THERAPY

Progression and application of CRISPR-Cas genomic editors

(2021) Li Yang et al.   METHODS

CRISPR prime editing with ribonucleoprotein complexes in zebrafish and primary human cells

(2021) Karl Petri et al.   NATURE BIOTECHNOLOGY

Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

(2021) Pengpeng Liu et al.   Nature Communications

Therapy Development by Genome Editing of Hematopoietic Stem Cells

(2021) Lola Koniali et al.   Cells

Correction of RNA splicing defect in β654-thalassemia mice using CRISPR/Cas9 gene-editing technology

(2021) Dan Lu et al.   HAEMATOLOGICA

Current status of beta‐thalassemia and its treatment strategies

(2021) Shaukat Ali et al.   Molecular Genetics & Genomic Medicine

Plant Prime Editors Enable Precise Gene Editing in Rice Cells

(2020) Xu Tang et al.   Molecular Plant

Prime genome editing in rice and wheat

(2020) Qiupeng Lin et al.   NATURE BIOTECHNOLOGY

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

Engineering herbicide resistance via prime editing in rice

(2020) Haroon Butt et al.   PLANT BIOTECHNOLOGY JOURNAL

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

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

A web tool for the design of prime-editing guide RNAs

(2020) Ryan D. Chow et al.   Nature Biomedical Engineering

Highly efficient therapeutic gene editing of human hematopoietic stem cells

(2019) Yuxuan Wu et al.   NATURE MEDICINE

Using Large Datasets to Understand Nanotechnology

(2019) Kalina Paunovska et al.   ADVANCED MATERIALS

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

(2019) Andrew V. Anzalone et al.   NATURE

Analyzing 2000 in Vivo Drug Delivery Data Points Reveals Cholesterol Structure Impacts Nanoparticle Delivery

(2018) Kalina Paunovska et al.   ACS Nano

Molecular basis of β thalassemia and potential therapeutic targets

(2018) Swee Lay Thein   BLOOD CELLS MOLECULES AND DISEASES

Recent Progress on Genetic Diagnosis and Therapy for β-Thalassemia in China and Around the World

(2018) Jingzhi Zhang et al.   HUMAN GENE THERAPY

A Direct Comparison of in Vitro and in Vivo Nucleic Acid Delivery Mediated by Hundreds of Nanoparticles Reveals a Weak Correlation

(2018) Kalina Paunovska et al.   NANO LETTERS

CRISPR/Cas9 genome editing in human hematopoietic stem cells

(2018) Rasmus O Bak et al.   Nature Protocols

CRISPOR: intuitive guide selection for CRISPR/Cas9 genome editing experiments and screens

(2018) Jean-Paul Concordet et al.   NUCLEIC ACIDS RESEARCH

Commentary: Programmable base editing of A·T to G·C in genomic DNA without DNA cleavage

(2018) Ianis G. Matsoukas   Frontiers in Genetics

Treatment of β654 -thalassaemia by TALENs in a mouse model

(2018) Yudan Fang et al.   CELL PROLIFERATION

Barcoding chemical modifications into nucleic acids improves drug stability in vivo

(2018) Cory D. Sago et al.   Journal of Materials Chemistry B

Nanoparticles That Deliver RNA to Bone Marrow Identified by in Vivo Directed Evolution

(2018) Cory D. Sago et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing

(2018) Cory D. Sago et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Barcoded nanoparticles for high throughput in vivo discovery of targeted therapeutics

(2017) James E. Dahlman et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Correction of β-thalassemia mutant by base editor in human embryos

(2017) Puping Liang et al.   Protein & Cell

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

(2016) Alexis C. Komor et al.   NATURE

BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis

(2015) Matthew C. Canver et al.   NATURE

Cas-OFFinder: a fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases

(2014) S. Bae et al.   BIOINFORMATICS

Therapeutic effects of induced pluripotent stem cells in chimeric mice with  -thalassemia

(2014) G. Yang et al.   HAEMATOLOGICA

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

Genetic association studies in  -hemoglobinopathies

(2013) S. L. Thein   Hematology-American Society of Hematology Education Program

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

Binding patterns of BCL11A in the globin and GATA1 loci and characterization of the BCL11A fetal hemoglobin locus

(2010) Kiran Jawaid et al.   BLOOD CELLS MOLECULES AND DISEASES

KLF1 regulates BCL11A expression and γ- to β-globin gene switching

(2010) Dewang Zhou et al.   NATURE GENETICS

Developmental and species-divergent globin switching are driven by BCL11A

(2009) Vijay G. Sankaran et al.   NATURE

Global epidemiology of haemoglobin disorders and derived service indicators

(2008) Bernadette Modell   BULLETIN OF THE WORLD HEALTH ORGANIZATION

A novel transgenic mouse model produced from lentiviral germline integration for the study of  -thalassemia gene therapy

(2008) W. Li et al.   HAEMATOLOGICA

Amelioration of β654-thalassemia in mouse model with the knockdown of aberrantly spliced β-globin mRNA

(2008) Shuyang Xie et al.   Journal of Genetics and Genomics

Human Fetal Hemoglobin Expression Is Regulated by the Developmental Stage-Specific RepressorBCL11A

(2008) Vijay G. Sankaran et al.   SCIENCE