Improved methods of AAV-mediated gene targeting for human cell lines using ribosome-skipping 2A peptide

Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant

(2014) Sandra P Melo et al.   MOLECULAR THERAPY

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

(2014) A. Barzel et al.   NATURE

A Comparative Analysis of Constitutive Promoters Located in Adeno-Associated Viral Vectors

(2014) Lkhagvasuren Damdindorj et al.   PLoS One

Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa

(2014) V. Sebastiano et al.   Science Translational Medicine

Cas9 as a versatile tool for engineering biology

(2013) Prashant Mali et al.   NATURE METHODS

Genome engineering using the CRISPR-Cas9 system

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Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs

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Simple Monitoring of Gene Targeting Efficiency in Human Somatic Cell Lines Using the PIGA Gene

(2012) Sivasundaram Karnan et al.   PLoS One

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

(2012) M. Jinek et al.   SCIENCE

2A to the Fore – Research, Technology and Applications

(2011) Garry A. Luke et al.   Biotechnology & Genetic Engineering Reviews

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

AAV-mediated gene targeting methods for human cells

(2011) Iram F Khan et al.   Nature Protocols

Mutation of a single allele of the cancer susceptibility gene BRCA1 leads to genomic instability in human breast epithelial cells

(2011) H. Konishi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

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Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome

(2010) R. C. DeKelver et al.   GENOME RESEARCH

Deregulation of the PI3K and KRAS signaling pathways in human cancer cells determines their response to everolimus

(2010) Federica Di Nicolantonio et al.   JOURNAL OF CLINICAL INVESTIGATION

Engineering of Human Pluripotent Stem Cells by AAV-mediated Gene Targeting

(2010) Iram F Khan et al.   MOLECULAR THERAPY

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

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

(2009) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Knockin of mutant PIK3CA activates multiple oncogenic pathways

(2009) John P. Gustin et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A panel of isogenic human cancer cells suggests a therapeutic approach for cancers with inactivated p53

(2009) S. Sur et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene Therapy Using Adeno-Associated Virus Vectors

(2008) S. Daya et al.   CLINICAL MICROBIOLOGY REVIEWS

Clinical gene therapy using recombinant adeno-associated virus vectors

(2008) C Mueller et al.   GENE THERAPY

Essential function of Chk1 can be uncoupled from DNA damage checkpoint and replication control

(2008) D. Wilsker et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Replacement of normal with mutant alleles in the genome of normal human cells unveils mutation-specific drug responses

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