Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery

Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications

(2016) Thomas Gaj et al.   MOLECULAR THERAPY

Genome-editing Technologies for Gene and Cell Therapy

(2016) Morgan L Maeder et al.   MOLECULAR THERAPY

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

(2016) Keiichiro Suzuki et al.   NATURE

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

(2016) Daniel P. Dever et al.   NATURE

In vitro and ex vivo strategies for intracellular delivery

(2016) Martin P. Stewart et al.   NATURE

Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease

(2016) Suk See De Ravin et al.   NATURE BIOTECHNOLOGY

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Digital PCR to assess gene-editing frequencies (GEF-dPCR) mediated by designer nucleases

(2016) Ulrike Mock et al.   Nature Protocols

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles

(2016) Ming Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Genome-Editing Technologies: Principles and Applications

(2016) Thomas Gaj et al.   Cold Spring Harbor Perspectives in Biology

Post-translational Regulation of Cas9 during G1 Enhances Homology-Directed Repair

(2016) Tony Gutschner et al.   Cell Reports

A Cas9 Variant for Efficient Generation of Indel-Free Knockin or Gene-Corrected Human Pluripotent Stem Cells

(2016) Sara E. Howden et al.   Stem Cell Reports

Viral Vectors for Gene Therapy: Translational and Clinical Outlook

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

Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining

(2015) Takeshi Maruyama et al.   NATURE BIOTECHNOLOGY

Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

(2015) Jianbin Wang et al.   NATURE BIOTECHNOLOGY

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells

(2015) Van Trung Chu et al.   NATURE BIOTECHNOLOGY

MicroRNA-based conversion of human fibroblasts into striatal medium spiny neurons

(2015) Michelle Richner et al.   Nature Protocols

Efficient delivery of nuclease proteins for genome editing in human stem cells and primary cells

(2015) Jia Liu et al.   Nature Protocols

Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery

(2015) Jianbin Wang et al.   NUCLEIC ACIDS RESEARCH

Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template

(2015) Blythe D. Sather et al.   Science Translational Medicine

Improved Cell-Penetrating Zinc-Finger Nuclease Proteins for Precision Genome Engineering

(2015) Jia Liu et al.   Molecular Therapy-Nucleic Acids

Cloning-free CRISPR

(2015) Mandana Arbab et al.   Stem Cell Reports

Protein Delivery Using Cys2–His2 Zinc-Finger Domains

(2014) Thomas Gaj et al.   ACS Chemical Biology

Genome Engineering with Targetable Nucleases

(2014) Dana Carroll   Annual Review of Biochemistry

Adeno-associated virus inverted terminal repeats stimulate gene editing

(2014) M L Hirsch   GENE THERAPY

Gene disruption by cell-penetrating peptide-mediated delivery of Cas9 protein and guide RNA

(2014) S. Ramakrishna et al.   GENOME RESEARCH

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

(2014) S. Kim et al.   GENOME RESEARCH

Isolation of single-base genome-edited human iPS cells without antibiotic selection

(2014) Yuichiro Miyaoka et al.   NATURE METHODS

Generation of Human Striatal Neurons by MicroRNA-Dependent Direct Conversion of Fibroblasts

(2014) Matheus B. Victor et al.   NEURON

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cell-Penetrating Peptide-Mediated Delivery of TALEN Proteins via Bioconjugation for Genome Engineering

(2014) Jia Liu et al.   PLoS One

Fibroblast growth factor-2 up-regulates the expression of nestin through the Ras–Raf–ERK–Sp1 signaling axis in C6 glioma cells

(2013) Kai-Wei Chang et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering

(2013) Sandrine Boissel et al.   NUCLEIC ACIDS RESEARCH

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

A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype

(2013) Brian L Ellis et al.   Virology Journal

An Inhibitor of Nonhomologous End-Joining Abrogates Double-Strand Break Repair and Impedes Cancer Progression

(2012) Mrinal Srivastava et al.   CELL

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

Targeted gene knockout by direct delivery of zinc-finger nuclease proteins

(2012) Thomas Gaj et al.   NATURE METHODS

Astrocytes regulate adult hippocampal neurogenesis through ephrin-B signaling

(2012) Randolph S Ashton et al.   NATURE NEUROSCIENCE

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

(2012) M. Jinek et al.   SCIENCE

Directed Evolution of Adeno-associated Virus for Enhanced Gene Delivery and Gene Targeting in Human Pluripotent Stem Cells

(2011) Prashanth Asuri et al.   MOLECULAR THERAPY

High-frequency genome editing using ssDNA oligonucleotides with zinc-finger nucleases

(2011) Fuqiang Chen et al.   NATURE METHODS

AAV-mediated gene targeting methods for human cells

(2011) Iram F Khan et al.   Nature Protocols

Regulation of Homologous Recombination in Eukaryotes

(2010) Wolf-Dietrich Heyer et al.   Annual Review of Genetics

Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology

(2010) Salvatore J. Orlando et al.   NUCLEIC ACIDS RESEARCH

Neural differentiation of human induced pluripotent stem cells follows developmental principles but with variable potency

(2010) B.-Y. Hu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene Targeting of a Disease-Related Gene in Human Induced Pluripotent Stem and Embryonic Stem Cells

(2009) Jizhong Zou et al.   Cell Stem Cell

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

(2009) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Regulation of DNA repair throughout the cell cycle

(2008) Dana Branzei et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases

(2008) Y. Santiago et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA