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Journal ArticleDOI

A library of TAL effector nucleases spanning the human genome

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TLDR
A genome-scale collection of TALENs for efficient and scalable gene targeting in human cells is presented and single- and double-gene-knockout cells in which NF-κB signaling pathways were disrupted showed unambiguous suppression of signal transduction.
Abstract
A collection of TALENs targeted to 18,740 human protein-coding genes will facilitate genetic engineering of human cells.

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Citations
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Development of Chimeric Cas9 Nucleases for Accurate and Flexible Genome Editing

TL;DR: Chimeric Cas9 platforms are developed and characterized to provide solutions to limitations of wild-type Cas9 nucleases and show improvements in mutagenesis efficacy and targeting feasibility.
Journal ArticleDOI

GRK5-Knockout Mice Generated by TALEN-Mediated Gene Targeting.

TL;DR: The generation of mice with genetic knockout (KO) of the G protein-coupled receptor kinase (GRK) 5 gene is investigated by microinjection of TALEN mRNA, a new type of engineered nuclease that is very effective for directed gene disruption in any genome sequence.
Journal ArticleDOI

Methods of genome engineering: a new era of molecular biology

TL;DR: This review discusses and compares the most important methods for gene inactivation and editing, as well as methods for incorporation of heterologous genes into the genome.
Book ChapterDOI

Designing and testing the activities of TAL effector nucleases.

TL;DR: A procedure to design active TALENs using a scoring system named Scoring Algorithm for Predicted TalEN Activity (SAPTA) and a method to test the activity of individual and pairs of TALens are described.
Journal ArticleDOI

Recent advances in developing molecular tools for targeted genome engineering of mammalian cells.

TL;DR: This minireview introduces the most recently spotlighted molecular genome engineering tools with their key features and ongoing modifications for better performance.
References
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Journal ArticleDOI

Initial sequencing and analysis of the human genome.

Eric S. Lander, +248 more
- 15 Feb 2001 - 
TL;DR: The results of an international collaboration to produce and make freely available a draft sequence of the human genome are reported and an initial analysis is presented, describing some of the insights that can be gleaned from the sequence.
Journal ArticleDOI

Multiplex Genome Engineering Using CRISPR/Cas Systems

TL;DR: The type II prokaryotic CRISPR (clustered regularly interspaced short palindromic repeats)/Cas adaptive immune system has been shown to facilitate RNA-guided site-specific DNA cleavage as discussed by the authors.
Journal ArticleDOI

The sequence of the human genome.

J. Craig Venter, +272 more
- 16 Feb 2001 - 
TL;DR: Comparative genomic analysis indicates vertebrate expansions of genes associated with neuronal function, with tissue-specific developmental regulation, and with the hemostasis and immune systems are indicated.

Multiplex Genome Engineering Using CRISPR/Cas Systems

TL;DR: Two different type II CRISPR/Cas systems are engineered and it is demonstrated that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells, demonstrating easy programmability and wide applicability of the RNA-guided nuclease technology.
Journal ArticleDOI

RNA-Guided Human Genome Engineering via Cas9

TL;DR: The type II bacterial CRISPR system is engineer to function with custom guide RNA (gRNA) in human cells to establish an RNA-guided editing tool for facile, robust, and multiplexable human genome engineering.
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