TY - JOUR
T1 - Advances in targeted genome editing
AU - Perez-Pinera, Pablo
AU - Ousterout, David G.
AU - Gersbach, Charles A.
N1 - Funding Information:
This work was supported by The Hartwell Foundation , a Basil O’Connor Starter Scholar Award from the March of Dimes, an NSF Faculty Early Career Development (CAREER) Award ( 1151035 ) and an NIH Director's New Innovator Award ( 1DP2OD008586 ).
PY - 2012/8
Y1 - 2012/8
N2 - New technologies have recently emerged that enable targeted editing of genomes in diverse systems. This includes precise manipulation of gene sequences in their natural chromosomal context and addition of transgenes to specific genomic loci. This progress has been facilitated by advances in engineering targeted nucleases with programmable, site-specific DNA-binding domains, including zinc finger proteins and transcription activator-like effectors (TALEs). Recent improvements have enhanced nuclease performance, accelerated nuclease assembly, and lowered the cost of genome editing. These advances are driving new approaches to many areas of biotechnology, including biopharmaceutical production, agriculture, creation of transgenic organisms and cell lines, and studies of genome structure, regulation, and function. Genome editing is also being investigated in preclinical and clinical gene therapies for many diseases.
AB - New technologies have recently emerged that enable targeted editing of genomes in diverse systems. This includes precise manipulation of gene sequences in their natural chromosomal context and addition of transgenes to specific genomic loci. This progress has been facilitated by advances in engineering targeted nucleases with programmable, site-specific DNA-binding domains, including zinc finger proteins and transcription activator-like effectors (TALEs). Recent improvements have enhanced nuclease performance, accelerated nuclease assembly, and lowered the cost of genome editing. These advances are driving new approaches to many areas of biotechnology, including biopharmaceutical production, agriculture, creation of transgenic organisms and cell lines, and studies of genome structure, regulation, and function. Genome editing is also being investigated in preclinical and clinical gene therapies for many diseases.
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U2 - 10.1016/j.cbpa.2012.06.007
DO - 10.1016/j.cbpa.2012.06.007
M3 - Review article
C2 - 22819644
AN - SCOPUS:84865297223
SN - 1367-5931
VL - 16
SP - 268
EP - 277
JO - Current Opinion in Chemical Biology
JF - Current Opinion in Chemical Biology
IS - 3-4
ER -