CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice

CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse

(2016) Yuting Guan et al.   EMBO Molecular Medicine

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

(2016) Daniel P. Dever et al.   NATURE

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

(2016) Benjamin P. Kleinstiver et al.   NATURE

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

(2015) R. Sharma et al.   BLOOD

Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C > T) mutation in β-thalassemia-derived iPSCs

(2015) Peng Xu et al.   Scientific Reports

Highly efficient RNA-guided genome editing in human cells via delivery of purified Cas9 ribonucleoproteins

(2014) S. Kim et al.   GENOME RESEARCH

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Genotyping with CRISPR-Cas-derived RNA-guided endonucleases

(2014) Jong Min Kim et al.   Nature Communications

Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

(2014) Steven Lin et al.   eLife

Robust ZFN-mediated genome editing in adult hemophilic mice

(2013) X. M. Anguela et al.   BLOOD

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

(2013) Gerald Schwank et al.   Cell Stem Cell

Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9

(2013) Yuxuan Wu et al.   Cell Stem Cell

Highly efficient gene knockout in mice and zebrafish with RNA-guided endonucleases

(2013) Y. H. Sung et al.   GENOME RESEARCH

Analysis of off-target effects of CRISPR/Cas-derived RNA-guided endonucleases and nickases

(2013) S. W. Cho et al.   GENOME RESEARCH

One-step generation of different immunodeficient mice with multiple gene modifications by CRISPR/Cas9 mediated genome engineering

(2013) Jiankui Zhou et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

An interactive mutation database for human coagulation factor IX provides novel insights into the phenotypes and genetics of hemophilia B

(2013) P. M. Rallapalli et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

RNA-programmed genome editing in human cells

(2013) Martin Jinek et al.   eLife

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Diagnostic guidelines for high-resolution melting curve (HRM) analysis: An interlaboratory validation ofBRCA1mutation scanning using the 96-well LightScanner™

(2009) Nienke van der Stoep et al.   HUMAN MUTATION

Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human

(2008) S E Khorsandi et al.   CANCER GENE THERAPY

Impact of non-homologous end-joining deficiency on random and targeted DNA integration: implications for gene targeting

(2008) Susumu Iiizumi et al.   NUCLEIC ACIDS RESEARCH