Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

(2019) Morgan L. Maeder et al.   NATURE MEDICINE

Delivering SaCas9 mRNA by lentivirus-like bionanoparticles for transient expression and efficient genome editing

(2019) Baisong Lu et al.   NUCLEIC ACIDS RESEARCH

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

Development of a CRISPR/Cas9-based therapy for Hutchinson–Gilford progeria syndrome

(2019) Olaya Santiago-Fernández et al.   NATURE MEDICINE

Single-dose CRISPR–Cas9 therapy extends lifespan of mice with Hutchinson–Gilford progeria syndrome

(2019) Ergin Beyret et al.   NATURE MEDICINE

Three-year follow-up of Phase 1 and 2a rAAV.sFLT-1 subretinal gene therapy trials for exudative age related macular degeneration

(2019) E.P. Rakoczy et al.   AMERICAN JOURNAL OF OPHTHALMOLOGY

Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations

(2019) Reza Shahbazi et al.   NATURE MATERIALS

Low risk to retina from sustained suppression of VEGF

(2019) Peter A. Campochiaro   JOURNAL OF CLINICAL INVESTIGATION

Anti-VEGF therapy: higher potency and long-lasting antagonism are not necessarily better

(2019) Ayumi Usui-Ouchi et al.   JOURNAL OF CLINICAL INVESTIGATION

Continuous evolution of base editors with expanded target compatibility and improved activity

(2019) Benjamin W. Thuronyi et al.   NATURE BIOTECHNOLOGY

Delivering Cas9/sgRNA ribonucleoprotein (RNP) by lentiviral capsid-based bionanoparticles for efficient ‘hit-and-run’ genome editing

(2019) Pin Lyu et al.   NUCLEIC ACIDS RESEARCH

Minimap2: pairwise alignment for nucleotide sequences

(2018) Heng Li   BIOINFORMATICS

Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements

(2018) Michael Kosicki et al.   NATURE BIOTECHNOLOGY

Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction

(2018) Luke W Koblan et al.   NATURE BIOTECHNOLOGY

p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells

(2018) Robert J. Ihry et al.   NATURE MEDICINE

Accurate detection of complex structural variations using single-molecule sequencing

(2018) Fritz J. Sedlazeck et al.   NATURE METHODS

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

(2018) Taeyoung Koo et al.   Nature Communications

High Efficiency Gene Correction in Hematopoietic Cells by Donor-Template-Free CRISPR/Cas9 Genome Editing

(2018) Duran Sürün et al.   Molecular Therapy-Nucleic Acids

VSV-G-Enveloped Vesicles for Traceless Delivery of CRISPR-Cas9

(2018) Claudia Montagna et al.   Molecular Therapy-Nucleic Acids

Time-Restricted PiggyBac DNA Transposition by Transposase Protein Delivery Using Lentivirus-Derived Nanoparticles

(2018) Kristian Alsbjerg Skipper et al.   Molecular Therapy-Nucleic Acids

Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

(2018) Bumwhee Lee et al.   Nature Biomedical Engineering

In vivo CRISPR editing with no detectable genome-wide off-target mutations

(2018) Pinar Akcakaya et al.   NATURE

Preclinical Evaluation of ADVM-022, a Novel Gene Therapy Approach to Treating Wet Age-Related Macular Degeneration

(2018) Ruslan Grishanin et al.   MOLECULAR THERAPY

High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population

(2018) Dimitrios L. Wagner et al.   NATURE MEDICINE

Genome editing in primary cells and in vivo using viral-derived Nanoblades loaded with Cas9-sgRNA ribonucleoproteins

(2018) Philippe E. Mangeot et al.   Nature Communications

A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing

(2018) Ang Li et al.   Molecular Therapy-Methods & Clinical Development

AAV2/8 Anti-angiogenic Gene Therapy Using Single-Chain Antibodies Inhibits Murine Choroidal Neovascularization

(2018) Chris P. Hughes et al.   Molecular Therapy-Methods & Clinical Development

Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration

(2017) Kyoungmi Kim et al.   GENOME RESEARCH

Lentiviral Vector Gene Transfer of Endostatin/Angiostatin for Macular Degeneration (GEM) Study

(2017) Peter A. Campochiaro et al.   HUMAN GENE THERAPY

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

(2017) Xue Gao et al.   NATURE

In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

(2017) Eunji Kim et al.   Nature Communications

In Vivo Knockout of the Vegfa Gene by Lentiviral Delivery of CRISPR/Cas9 in Mouse Retinal Pigment Epithelium Cells

(2017) Andreas Holmgaard et al.   Molecular Therapy-Nucleic Acids

Integrase-Deficient Lentiviral Vector as an All-in-One Platform for Highly Efficient CRISPR/Cas9-Mediated Gene Editing

(2017) Pavel I. Ortinski et al.   Molecular Therapy-Methods & Clinical Development

Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair

(2017) Kunwoo Lee et al.   Nature Biomedical Engineering

Cas-analyzer: an online tool for assessing genome editing results using NGS data

(2016) Jeongbin Park et al.   BIOINFORMATICS

Lentiviral Delivery of Proteins for Genome Engineering

(2016) Yujia Cai et al.   CURRENT GENE THERAPY

Lentivirus pre-packed with Cas9 protein for safer gene editing

(2016) J G Choi et al.   GENE THERAPY

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

(2016) Daniel P. Dever et al.   NATURE

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Lentiviral vectors, two decades later

(2016) L. Naldini et al.   SCIENCE

CRISPR/Cas9-Mediated Genome Editing of Herpesviruses Limits Productive and Latent Infections

(2016) Ferdy R. van Diemen et al.   PLoS Pathogens

Aquaporin 4-specific T cells and NMO-IgG cause primary retinal damage in experimental NMO/SD

(2016) Bleranda Zeka et al.   Acta Neuropathologica Communications

Adenovirus-Mediated Somatic Genome Editing ofPtenby CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses

(2015) Dan Wang et al.   HUMAN GENE THERAPY

Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial

(2015) Elizabeth P Rakoczy et al.   LANCET

Highly efficient in vitro and in vivo delivery of functional RNAs using new versatile MS2-chimeric retrovirus-like particles

(2015) Anne Prel et al.   Molecular Therapy-Methods & Clinical Development

CCTop: An Intuitive, Flexible and Reliable CRISPR/Cas9 Target Prediction Tool

(2015) Manuel Stemmer et al.   PLoS One

Optimization of an Image-Guided Laser-Induced Choroidal Neovascularization Model in Mice

(2015) Yan Gong et al.   PLoS One

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

(2014) S. Kim et al.   GENOME RESEARCH

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Improved vectors and genome-wide libraries for CRISPR screening

(2014) Neville E Sanjana et al.   NATURE METHODS

Easy quantitative assessment of genome editing by sequence trace decomposition

(2014) Eva K. Brinkman et al.   NUCLEIC ACIDS RESEARCH

Life of psi: How full-length HIV-1 RNAs become packaged genomes in the viral particles

(2014) Malika Kuzembayeva et al.   VIROLOGY

Delivering the second revolution in site-specific nucleases

(2014) Perry B Hackett et al.   eLife

Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases

(2014) Yujia Cai et al.   eLife

DNA transposition by protein transduction of the piggyBac transposase from lentiviral Gag precursors

(2013) Yujia Cai et al.   NUCLEIC ACIDS RESEARCH

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

(2013) F. Mingozzi et al.   Science Translational Medicine

Targeted deletion of Vegfa in adult mice induces vision loss

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

Age-related macular degeneration

(2012) Laurence S Lim et al.   LANCET

BEDTools: a flexible suite of utilities for comparing genomic features

(2010) Aaron R. Quinlan et al.   BIOINFORMATICS

Toxicity and cellular uptake of gold nanoparticles: what we have learned so far?

(2010) Alaaldin M. Alkilany et al.   JOURNAL OF NANOPARTICLE RESEARCH

Protein transduction from retroviral Gag precursors

(2010) Christine Voelkel et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Inhibition of Choroidal Neovascularization via Brief Subretinal Exposure to a Newly Developed Lentiviral Vector Pseudotyped with Sendai Viral Envelope Proteins

(2009) Yusuke Murakami et al.   HUMAN GENE THERAPY

Hybrid Lentivirus-transposon Vectors With a Random Integration Profile in Human Cells

(2009) Nicklas H Staunstrup et al.   MOLECULAR THERAPY