CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration

Retinal Gene Therapy: Surgical Vector Delivery in the Translation to Clinical Trials

(2017) G. Alex Ochakovski et al.   Frontiers in Neuroscience

Cpf1 nucleases demonstrate robust activity to induce DNA modification by exploiting homology directed repair pathways in mammalian cells

(2016) Eszter Tóth et al.   Biology Direct

Therapeutic applications of CRISPR RNA-guided genome editing

(2016) Taeyoung Koo et al.   Briefings in Functional Genomics

Genome-wide target specificities of CRISPR-Cas9 nucleases revealed by multiplex Digenome-seq

(2016) Daesik Kim et al.   GENOME RESEARCH

The CRISPR-associated DNA-cleaving enzyme Cpf1 also processes precursor CRISPR RNA

(2016) Ines Fonfara et al.   NATURE

Targeted mutagenesis in mice by electroporation of Cpf1 ribonucleoproteins

(2016) Junho K Hur et al.   NATURE BIOTECHNOLOGY

Multiplex gene editing by CRISPR–Cpf1 using a single crRNA array

(2016) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

Genome-wide specificities of CRISPR-Cas Cpf1 nucleases in human cells

(2016) Benjamin P Kleinstiver et al.   NATURE BIOTECHNOLOGY

Genome-wide analysis reveals specificities of Cpf1 endonucleases in human cells

(2016) Daesik Kim et al.   NATURE BIOTECHNOLOGY

In vivo high-throughput profiling of CRISPR–Cpf1 activity

(2016) Hui K Kim et al.   NATURE METHODS

Cornerstones of CRISPR–Cas in drug discovery and therapy

(2016) Christof Fellmann et al.   NATURE REVIEWS DRUG DISCOVERY

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

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

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

(2015) Bernd Zetsche et al.   CELL

Erratum: Defining response to anti-VEGF therapies in neovascular AMD

(2015) W M Amoaku et al.   EYE

Local and systemic responses following intravitreous injection of AAV2-encoded modified Volvox channelrhodopsin-1 in a genetically blind rat model

(2015) E Sugano et al.   GENE THERAPY

The Expression Pattern of Systemically Injected AAV9 in the Developing Mouse Retina Is Determined by Age

(2015) Leah C Byrne et al.   MOLECULAR THERAPY

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

(2015) Daesik Kim et al.   NATURE METHODS

Isaac: ultra-fast whole-genome secondary analysis on Illumina sequencing platforms

(2013) Come Raczy et al.   BIOINFORMATICS

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

Age-Related Macular Degeneration

(2013) Lily K. Cheung et al.   PHARMACOTHERAPY

A Single Intravenous AAV9 Injection Mediates Bilateral Gene Transfer to the Adult Mouse Retina

(2013) Alexis-Pierre Bemelmans et al.   PLoS One

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Therapies for Neovascular Age-Related Macular Degeneration: Current Approaches and Pharmacologic Agents in Development

(2013) Mostafa Hanout et al.   Biomed Research International

RNA-programmed genome editing in human cells

(2013) Martin Jinek et al.   eLife

Targeted deletion of Vegfa in adult mice induces vision loss

(2012) Toshihide Kurihara et al.   JOURNAL OF CLINICAL INVESTIGATION

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

(2012) M. Jinek et al.   SCIENCE

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

(2011) Amit C Nathwani et al.   MOLECULAR THERAPY

Vascular endothelial growth factor and age-related macular degeneration: from basic science to therapy

(2010) Napoleone Ferrara   NATURE MEDICINE

AQUEOUS VASCULAR ENDOTHELIAL GROWTH FACTOR AFTER INTRAVITREAL INJECTION OF PEGAPTANIB OR RANIBIZUMAB IN PATIENTS WITH AGE-RELATED MACULAR DEGENERATION

(2010) Osamu Sawada et al.   RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES

AAV-mediated Local Delivery of Interferon-β for the Treatment of Retinoblastoma in Preclinical Models

(2009) Chie-Schin Shih et al.   NEUROMOLECULAR MEDICINE

Neovascular Age-related Macular Degeneration: Intraocular Cytokines and Growth Factors and the Influence of Therapy with Ranibizumab

(2009) Marion Funk et al.   OPHTHALMOLOGY

Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE