Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins

In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice

(2021) Luke W. Koblan et al.   NATURE

Quantitative evaluation of chromosomal rearrangements in gene-edited human stem cells by CAST-Seq

(2021) Giandomenico Turchiano et al.   Cell Stem Cell

Adenine Base Editor Ribonucleoproteins Delivered by Lentivirus-Like Particles Show High On-Target Base Editing and Undetectable RNA Off-Target Activities

(2021) Pin Lyu et al.   CRISPR Journal

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

(2021) Kiran Musunuru et al.   NATURE

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

(2021) Tanja Rothgangl et al.   NATURE BIOTECHNOLOGY

Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing

(2021) Mitchell L. Leibowitz et al.   NATURE GENETICS

In vivo somatic cell base editing and prime editing

(2021) Gregory A. Newby et al.   MOLECULAR THERAPY

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

(2021) Gregory A. Newby et al.   NATURE

RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy

(2021) Andrea Sodi et al.   Orphanet Journal of Rare Diseases

Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering

(2021) Jennifer R. Hamilton et al.   Cell Reports

High-purity production and precise editing of DNA base editing ribonucleoproteins

(2021) Hyeon-Ki Jang et al.   Science Advances

Enhanced prime editing systems by manipulating cellular determinants of editing outcomes

(2021) Peter J. Chen et al.   CELL

The promise and challenge of therapeutic genome editing

(2020) Jennifer A. Doudna   NATURE

Evaluation and minimization of Cas9-independent off-target DNA editing by cytosine base editors

(2020) Jordan L. Doman et al.   NATURE BIOTECHNOLOGY

CRISPR-engineered T cells in patients with refractory cancer

(2020) Edward A. Stadtmauer et al.   SCIENCE

Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

(2020) Jonathan M. Levy et al.   Nature Biomedical Engineering

CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors

(2020) Dan Wang et al.   CELL

Directed evolution of adenine base editors with increased activity and therapeutic application

(2020) Nicole M. Gaudelli et al.   NATURE BIOTECHNOLOGY

Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity

(2020) Michelle F. Richter et al.   NATURE BIOTECHNOLOGY

Therapeutic base editing of human hematopoietic stem cells

(2020) Jing Zeng et al.   NATURE MEDICINE

Cytosine base editors with minimized unguided DNA and RNA off-target events and high on-target activity

(2020) Yi Yu et al.   Nature Communications

Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping

(2020) Peter Gee et al.   Nature Communications

Maximizing lentiviral vector gene transfer in the CNS

(2020) Morgane Humbel et al.   GENE THERAPY

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

(2020) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

CHANGE-seq reveals genetic and epigenetic effects on CRISPR–Cas9 genome-wide activity

(2020) Cicera R. Lazzarotto et al.   NATURE BIOTECHNOLOGY

Highly efficient ‘hit-and-run’ genome editing with unconcentrated lentivectors carrying Vpr.Prot.Cas9 protein produced from RRE-containing transcripts

(2020) Ivana Indikova et al.   NUCLEIC ACIDS RESEARCH

In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness

(2020) Wei-Hsi Yeh et al.   Science Translational Medicine

Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

(2020) Tuo Wei et al.   Nature Communications

Large-Fragment Deletions Induced by Cas9 Cleavage while Not in the BEs System

(2020) Yuning Song et al.   Molecular Therapy-Nucleic Acids

Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing

(2020) Susie Suh et al.   Nature Biomedical Engineering

CRISPResso2 provides accurate and rapid genome editing sequence analysis

(2019) Kendell Clement et al.   NATURE BIOTECHNOLOGY

Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors

(2019) Tony P. Huang 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

Base editor correction of COL7A1 in recessive dystrophic epidermolysis bullosa patient-derived fibroblasts and iPSCs

(2019) Mark J. Osborn et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Intact Viral Particle Counts Measured by Flow Virometry Provides Insight into the Infectivity and Genome Packaging Efficiency of the Moloney Murine Leukemia Virus

(2019) Tyler Milston Renner et al.   JOURNAL OF VIROLOGY

Highly efficient multiplex human T cell engineering without double-strand breaks using Cas9 base editors

(2019) Beau R. Webber et al.   Nature Communications

Drug-Induced Liver Injury: Biomarkers, Requirements, Candidates, and Validation

(2019) Lucy Meunier et al.   Frontiers in Pharmacology

Mass spectrometry analysis reveals differences in the host cell protein species found in pseudotyped lentiviral vectors

(2018) Sabine Johnson et al.   BIOLOGICALS

UDiTaS™, a genome editing detection method for indels and genome rearrangements

(2018) Georgia Giannoukos et al.   BMC GENOMICS

Clinical use of lentiviral vectors

(2018) Michael C. Milone et al.   LEUKEMIA

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

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

(2018) Eugenio Mercuri et al.   NEW ENGLAND JOURNAL OF MEDICINE

In vivo base editing of post-mitotic sensory cells

(2018) Wei-Hsi Yeh et al.   Nature Communications

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

(2018) Pinar Akcakaya et al.   NATURE

Gesicle-Mediated Delivery of CRISPR/Cas9 Ribonucleoprotein Complex for Inactivating the HIV Provirus

(2018) Lee A. Campbell et al.   MOLECULAR THERAPY

Base editing: precision chemistry on the genome and transcriptome of living cells

(2018) Holly A. Rees et al.   NATURE REVIEWS GENETICS

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

Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models

(2017) Randy J. Chandler et al.   HUMAN GENE THERAPY

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR–Cas9 nuclease off-targets

(2017) Shengdar Q Tsai et al.   NATURE METHODS

Improving the DNA specificity and applicability of base editing through protein engineering and protein delivery

(2017) Holly A. Rees et al.   Nature Communications

Lentivirus pre-packed with Cas9 protein for safer gene editing

(2016) J G Choi et al.   GENE THERAPY

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

Targeted nucleotide editing using hybrid prokaryotic and vertebrate adaptive immune systems

(2016) K. Nishida et al.   SCIENCE

Viral Vectors: The Road to Reducing Genotoxicity

(2016) Rhiannon M. David et al.   TOXICOLOGICAL SCIENCES

Small molecule–triggered Cas9 protein with improved genome-editing specificity

(2015) Kevin M Davis et al.   Nature Chemical Biology

Lentiviral vectors as tools to understand central nervous system biology in mammalian model organisms

(2015) Louise C. Parr-Brownlie et al.   Frontiers in Molecular Neuroscience

MLV based viral-like-particles for delivery of toxic proteins and nuclear transcription factors

(2014) Dai-Tze Wu et al.   BIOMATERIALS

Retinal transduction profiles by high-capacity viral vectors

(2014) A Puppo et al.   GENE THERAPY

In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

(2013) Kevin Fitzgerald et al.   LANCET

Selective Neural Pathway Targeting Reveals Key Roles of Thalamostriatal Projection in the Control of Visual Discrimination

(2011) S. Kato et al.   JOURNAL OF NEUROSCIENCE

Protein Transfer Into Human Cells by VSV-G-induced Nanovesicles

(2011) Philippe-Emmanuel Mangeot et al.   MOLECULAR THERAPY

Protein delivery using engineered virus-like particles

(2011) S. J. Kaczmarczyk et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Importance of Membrane Structural Integrity for RPE65 Retinoid Isomerization Activity

(2010) Marcin Golczak et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Protein transduction from retroviral Gag precursors

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

Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy

(2010) Artur V. Cideciyan   PROGRESS IN RETINAL AND EYE RESEARCH

Ocular immune privilege

(2009) A W Taylor   EYE

Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation

(2009) Dan Wang et al.   MOLECULAR GENETICS AND METABOLISM

Leber congenital amaurosis: Genes, proteins and disease mechanisms

(2008) Anneke I. den Hollander et al.   PROGRESS IN RETINAL AND EYE RESEARCH