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 …
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 …
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 …
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 …
discussed as an attractive alternative to existing control measures because of their potential …
megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering
Rare-cleaving endonucleases have emerged as important tools for making targeted
genome modifications. While multiple platforms are now available to generate reagents for …
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 …
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?
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 …
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 …
(genome editing) has been a powerful research tool. In the past decade, the tools and …
Homing endonucleases: DNA scissors on a mission
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 …
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 …
both their own genes and their local genetic environment. The mechanisms that govern the …