Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy

G Silva, L Poirot, R Galetto, J Smith… - Current gene …, 2011 - ingentaconnect.com
The importance of safer approaches for gene therapy has been underscored by a series of
severe adverse events (SAEs) observed in patients involved in clinical trials for Severe …

Homing endonucleases: from microbial genetic invaders to reagents for targeted DNA modification

BL Stoddard - Structure, 2011 - cell.com
Homing endonucleases are microbial DNA-cleaving enzymes that mobilize their own
reading frames by generating double strand breaks at specific genomic invasion sites …

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

BD Sather, GS Romano Ibarra, K Sommer… - Science translational …, 2015 - science.org
Genetic mutations or engineered nucleases that disrupt the HIV co-receptor CCR5 block HIV
infection of CD4+ T cells. These findings have motivated the engineering of CCR5-specific …

A synthetic homing endonuclease-based gene drive system in the human malaria mosquito

N Windbichler, M Menichelli, PA Papathanos… - Nature, 2011 - nature.com
Genetic methods of manipulating or eradicating disease vector populations have long been
discussed as an attractive alternative to existing control measures because of their potential …

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

S Boissel, J Jarjour, A Astrakhan, A Adey… - Nucleic acids …, 2014 - academic.oup.com
Rare-cleaving endonucleases have emerged as important tools for making targeted
genome modifications. While multiple platforms are now available to generate reagents for …

Homing endonucleases from mobile group I introns: discovery to genome engineering

BL Stoddard - Mobile DNA, 2014 - Springer
Homing endonucleases are highly specific DNA cleaving enzymes that are encoded within
genomes of all forms of microbial life including phage and eukaryotic organelles. These …

Gene drive for mosquito control: where did it come from and where are we headed?

VM Macias, JR Ohm, JL Rasgon - International journal of environmental …, 2017 - mdpi.com
Mosquito-borne pathogens place an enormous burden on human health. The existing toolkit
is insufficient to support ongoing vector-control efforts towards meeting disease elimination …

Genome editing: a new approach to human therapeutics

M Porteus - Annual review of pharmacology and toxicology, 2016 - annualreviews.org
The ability to manipulate the genome with precise spatial and nucleotide resolution
(genome editing) has been a powerful research tool. In the past decade, the tools and …

Homing endonucleases: DNA scissors on a mission

M Hafez, G Hausner - Genome, 2012 - cdnsciencepub.com
Buried within the genomes of many microorganisms are genetic elements that encode rare-
cutting homing endonucleases that assist in the mobility of the elements that encode them …

Homing endonucleases: from genetic anomalies to programmable genomic clippers

M Belfort, RP Bonocora - Homing Endonucleases: Methods and Protocols, 2014 - Springer
Homing endonucleases are strong drivers of genetic exchange and horizontal transfer of
both their own genes and their local genetic environment. The mechanisms that govern the …