Viral Delivery Systems for CRISPR

Delivering CRISPR: a review of the challenges and approaches

(2018) Christopher A. Lino et al.   DRUG DELIVERY

Triple Vectors Expand AAV Transfer Capacity in the Retina

(2018) Andrea Maddalena et al.   MOLECULAR THERAPY

CRISPR-Cas12a target binding unleashes indiscriminate single-stranded DNase activity

(2018) Janice S. Chen et al.   SCIENCE

Direct observation of DNA target searching and cleavage by CRISPR-Cas12a

(2018) Yongmoon Jeon et al.   Nature Communications

Structural Basis for the Canonical and Non-canonical PAM Recognition by CRISPR-Cpf1

(2017) Takashi Yamano et al.   MOLECULAR CELL

Extending the Host Range of Bacteriophage Particles for DNA Transduction

(2017) Ido Yosef et al.   MOLECULAR CELL

Engineered Cpf1 variants with altered PAM specificities

(2017) Linyi Gao et al.   NATURE BIOTECHNOLOGY

Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models

(2017) Nicola Brunetti-Pierri et al.   VIRUS GENES

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

(2017) Eunji Kim et al.   Nature Communications

CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors

(2017) Rasmus O. Bak et al.   Cell Reports

Effect of PCSK9 Inhibitors on Clinical Outcomes in Patients With Hypercholesterolemia: A Meta‐Analysis of 35 Randomized Controlled Trials

(2017) Aris Karatasakis et al.   Journal of the American Heart Association

Highly efficient gene inactivation by adenoviral CRISPR/Cas9 in human primary cells

(2017) Olaf Voets et al.   PLoS One

Targeted Gene Manipulation in Plants Using the CRISPR/Cas Technology

(2016) Dandan Zhang et al.   Journal of Genetics and Genomics

CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

(2016) Li Xu et al.   MOLECULAR THERAPY

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

(2016) Daesik Kim et al.   NATURE BIOTECHNOLOGY

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations

(2016) Ignazio Maggio et al.   NUCLEIC ACIDS RESEARCH

Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells

(2016) Ignazio Maggio et al.   Scientific Reports

Viral Vectors for Gene Therapy: Translational and Clinical Outlook

(2015) Melissa A. Kotterman et al.   Annual Review of Biomedical Engineering

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

(2015) Bernd Zetsche et al.   CELL

Nanoparticle-based technologies for retinal gene therapy

(2015) Jeffrey Adijanto et al.   EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS

Potential pitfalls of CRISPR/Cas9-mediated genome editing

(2015) Rongxue Peng et al.   FEBS Journal

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

Inhibition of HIV-1 infection of primary CD4+ T-cells by gene editing of CCR5 using adenovirus-delivered CRISPR/Cas9

(2015) Chang Li et al.   JOURNAL OF GENERAL VIROLOGY

Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia

(2015) Eyal Banin et al.   MOLECULAR THERAPY

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Unbiased detection of off-target cleavage by CRISPR-Cas9 and TALENs using integrase-defective lentiviral vectors

(2015) Xiaoling Wang et al.   NATURE BIOTECHNOLOGY

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

(2015) Daesik Kim et al.   NATURE METHODS

Temperate and lytic bacteriophages programmed to sensitize and kill antibiotic-resistant bacteria

(2015) Ido Yosef et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A CRISPR-Based Screen Identifies Genes Essential for West-Nile-Virus-Induced Cell Death

(2015) Hongming Ma et al.   Cell Reports

Gene Therapy of ABCA4-Associated Diseases

(2015) A. Auricchio et al.   Cold Spring Harbor Perspectives in Medicine

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system

(2014) Danilo Maddalo et al.   NATURE

DNA interrogation by the CRISPR RNA-guided endonuclease Cas9

(2014) Samuel H. Sternberg et al.   NATURE

Sequence-specific antimicrobials using efficiently delivered RNA-guided nucleases

(2014) Robert J Citorik et al.   NATURE BIOTECHNOLOGY

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

(2014) Lukasz Swiech et al.   NATURE BIOTECHNOLOGY

Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing

(2014) Dirk Heckl et al.   NATURE BIOTECHNOLOGY

Exploiting CRISPR-Cas nucleases to produce sequence-specific antimicrobials

(2014) David Bikard et al.   NATURE BIOTECHNOLOGY

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

Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector

(2014) Ami M. Kabadi et al.   NUCLEIC ACIDS RESEARCH

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

(2013) V S Lopes et al.   GENE THERAPY

Triple Trans-Splicing Adeno-Associated Virus Vectors Capable of Transferring the Coding Sequence for Full-Length Dystrophin Protein into Dystrophic Mice

(2013) Taeyoung Koo et al.   HUMAN GENE THERAPY

Genome-wide recessive genetic screening in mammalian cells with a lentiviral CRISPR-guide RNA library

(2013) Hiroko Koike-Yusa et al.   NATURE BIOTECHNOLOGY

A largely random AAV integration profile after LPLD gene therapy

(2013) Christine Kaeppel et al.   NATURE MEDICINE

Nucleotide-resolution DNA double-strand break mapping by next-generation sequencing

(2013) Nicola Crosetto et al.   NATURE METHODS

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Phylogeny of Cas9 determines functional exchangeability of dual-RNA and Cas9 among orthologous type II CRISPR-Cas systems

(2013) Ines Fonfara et al.   NUCLEIC ACIDS RESEARCH

Protospacer recognition motifs

(2013) Shiraz A. Shah et al.   RNA Biology

Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

(2013) Ophir Shalem et al.   SCIENCE

Genetic Screens in Human Cells Using the CRISPR-Cas9 System

(2013) Tim Wang et al.   SCIENCE

DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice

(2012) Zongchao Han et al.   JOURNAL OF CLINICAL INVESTIGATION

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

(2012) M. Jinek et al.   SCIENCE

Phage Therapy: Concept to Cure

(2012) Eric C. Keen   Frontiers in Microbiology

State-of-the-Art Lentiviral Vectors for Research Use: Risk Assessment and Biosafety Recommendations

(2009) Katia Pauwels et al.   CURRENT GENE THERAPY

A 16-bp RBE element mediated Rep-dependent site-specific integration in AAVS1 transgenic mice for expression of hFIX

(2009) Z X Xu et al.   GENE THERAPY

A Hybrid Vector System Expands Adeno-associated Viral Vector Packaging Capacity in a Transgene-independent Manner

(2007) Arkasubhra Ghosh et al.   MOLECULAR THERAPY