CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice

AAV Infection: Protection from Cancer

(2017) Arun Srivastava et al.   HUMAN GENE THERAPY

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

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

(2016) Keiichiro Suzuki et al.   NATURE

Correction of the auditory phenotype in C57BL/6N mice via CRISPR/Cas9-mediated homology directed repair

(2016) Joffrey Mianné et al.   Genome Medicine

CRISPR-Cas9 for in vivo Gene Therapy: Promise and Hurdles

(2016) Wei-Jing Dai et al.   Molecular Therapy-Nucleic Acids

CRISPR-Cas: New Tools for Genetic Manipulations from Bacterial Immunity Systems

(2015) Wenyan Jiang et al.   Annual Review of Microbiology

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

(2015) R. Sharma et al.   BLOOD

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

Adeno-associated virus serotypes for gene therapeutics

(2015) Leszek Lisowski et al.   CURRENT OPINION IN PHARMACOLOGY

Inhibition of nonhomologous end joining to increase the specificity of CRISPR/Cas9 genome editing

(2015) Supriya V. Vartak et al.   FEBS Journal

New approaches to gene and cell therapy for hemophilia

(2015) T. Ohmori et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Don’t edit the human germ line

(2015) Edward Lanphier et al.   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining

(2015) Takeshi Maruyama et al.   NATURE BIOTECHNOLOGY

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells

(2015) Van Trung Chu et al.   NATURE BIOTECHNOLOGY

An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia

(2015) Alfica Sehgal et al.   NATURE MEDICINE

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

CRISPR/Cas9 for genome editing: progress, implications and challenges

(2014) F. Zhang et al.   HUMAN MOLECULAR GENETICS

Mechanisms and principles of homology search during recombination

(2014) Jörg Renkawitz et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

(2014) C. Long et al.   SCIENCE

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Robust ZFN-mediated genome editing in adult hemophilic mice

(2013) X. M. Anguela et al.   BLOOD

The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals

(2013) Jun Mimuro et al.   JOURNAL OF MEDICAL VIROLOGY

High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity

(2013) Vikram Pattanayak et al.   NATURE BIOTECHNOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Cas9 as a versatile tool for engineering biology

(2013) Prashant Mali et al.   NATURE METHODS

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

The CDC Hemophilia B mutation project mutation list: a new online resource

(2013) Tengguo Li et al.   Molecular Genetics & Genomic Medicine

The AAV Vector Toolkit: Poised at the Clinical Crossroads

(2012) Aravind Asokan et al.   MOLECULAR THERAPY

Minimizing the Inhibitory Effect of Neutralizing Antibody for Efficient Gene Expression in the Liver With Adeno-associated Virus 8 Vectors

(2012) Jun Mimuro et al.   MOLECULAR THERAPY

Hepatic Gene Transfer in Neonatal Mice by Adeno-Associated Virus Serotype 8 Vector

(2011) Lili Wang et al.   HUMAN GENE THERAPY

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

(2011) Hojun Li et al.   NATURE

A modified thrombin generation test for investigating very low levels of factor VIII activity in hemophilia A

(2009) Tomoko Matsumoto et al.   INTERNATIONAL JOURNAL OF HEMATOLOGY