Towards personalised allele-specific CRISPR gene editing to treat autosomal dominant disorders

The Human Gene Mutation Database: towards a comprehensive repository of inherited mutation data for medical research, genetic diagnosis and next-generation sequencing studies

(2017) Peter D. Stenson et al.   HUMAN GENETICS

Engineered Cpf1 variants with altered PAM specificities

(2017) Linyi Gao et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

Pathogenesis and treatments of TGFBI corneal dystrophies

(2016) Kyung Eun Han et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Exploiting the CRISPR/Cas9 PAM Constraint for Single-Nucleotide Resolution Interventions

(2016) Yi Li et al.   PLoS One

CRISPR/Cas9 DNA cleavage at SNP-derived PAM enables both in vitro and in vivo KRT12 mutation-specific targeting

(2015) D G Courtney et al.   GENE THERAPY

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Orthogonal gene knockout and activation with a catalytically active Cas9 nuclease

(2015) James E Dahlman et al.   NATURE BIOTECHNOLOGY

Cas9 gRNA engineering for genome editing, activation and repression

(2015) Samira Kiani et al.   NATURE METHODS

Structure and specificity of the RNA-guided endonuclease Cas9 during DNA interrogation, target binding and cleavage

(2015) Eric A. Josephs et al.   NUCLEIC ACIDS RESEARCH

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker et al.   SCIENCE

Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA

(2014) Hiroshi Nishimasu et al.   CELL

DNA interrogation by the CRISPR RNA-guided endonuclease Cas9

(2014) Samuel H. Sternberg et al.   NATURE

Genome-wide binding of the CRISPR endonuclease Cas9 in mammalian cells

(2014) Xuebing Wu et al.   NATURE BIOTECHNOLOGY

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

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

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

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

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

RNA-guided editing of bacterial genomes using CRISPR-Cas systems

(2013) Wenyan Jiang et al.   NATURE BIOTECHNOLOGY

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

(2012) M. Jinek et al.   SCIENCE

Development of Allele-Specific Therapeutic siRNA for Keratin 5 Mutations in Epidermolysis Bullosa Simplex

(2011) Sarah D. Atkinson et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

IC3D-Klassifikation von Hornhautdystrophien

(2011) J. Weiss et al.   KLINISCHE MONATSBLATTER FUR AUGENHEILKUNDE

Development of Allele-Specific Therapeutic siRNA in Meesmann Epithelial Corneal Dystrophy

(2011) Haihui Liao et al.   PLoS One

Clinical Findings and Treatments of Granular Corneal Dystrophy Type 2 (Avellino Corneal Dystrophy): A Review of the Literature

(2010) Kyung Eun Han et al.   Eye & Contact Lens-Science and Clinical Practice

Prevalence of Granular Corneal Dystrophy Type 2 (Avellino Corneal Dystrophy) in the Korean Population

(2010) Jae Hwan Lee et al.   OPHTHALMIC EPIDEMIOLOGY

Genetics of Anterior and Stromal Corneal Dystrophies

(2008) Vasilliki Poulaki et al.   Seminars in Ophthalmology