Generation of Fibroblasts Lacking the Sal-like 1 Gene by Using Transcription Activator-like Effector Nuclease-mediated Homologous Recombination [PDF]
The Sal-like 1 gene (Sall1) is essential for kidney development, and mutations in this gene result in abnormalities in the kidneys. Mice lacking Sall1 show agenesis or severe dysgenesis of the kidneys.
Se Eun Kim+5 more
doaj +4 more sources
Transcription activator-like effector nuclease-mediated transduction of exogenous gene into IL2RG locus. [PDF]
X-linked severe combined immunodeficiency (SCID-X1) caused by mutations in interleukin 2 receptor gamma (IL2RG) gene threatens the survival of affected boys during the first year of life unless hematopoietic stem cell transplantation is provided. Although viral vector-mediated gene therapy has been successfully performed in patients with no HLA-matched
Matsubara Y+6 more
europepmc +5 more sources
Efficient Gene Editing in Pluripotent Stem Cells by Bacterial Injection of Transcription Activator-Like Effector Nuclease Proteins. [PDF]
Abstract The type III secretion system (T3SS) of Pseudomonas aeruginosa is a powerful tool for direct protein delivery into mammalian cells and has successfully been used to deliver various exogenous proteins into mammalian cells.
Jia J+8 more
europepmc +5 more sources
Targeted gene disruption by use of transcription activator-like effector nuclease (TALEN) in the water flea Daphnia pulex. [PDF]
The cosmopolitan microcrustacean Daphnia pulex provides a model system for both human health research and monitoring ecosystem integrity. It is the first crustacean to have its complete genome sequenced, an unprecedented ca. 36% of which has no known homologs with any other species. Moreover, D.
Hiruta C+6 more
europepmc +7 more sources
Disruption of <i>CYP88B1</i> by transcription activator-like effector nuclease in potato and potential use to produce useful saponins. [PDF]
Potatoes produce steroidal glycoalkaloids (SGAs), toxic secondary metabolites associated with food poisoning. SGAs are synthesized by multiple biosynthetic enzymes. Knockdown of the CYP88B1 gene, also known as PGA3 or GAME4, is predicted to reduce toxic SGAs and accumulate steroidal saponins. These saponins not only serve as a source of steroidal drugs
Yasumoto S+7 more
europepmc +4 more sources
Comparing Zinc Finger Nucleases and Transcription Activator-Like Effector Nucleases for Gene Targeting in Drosophila [PDF]
AbstractZinc-finger nucleases have proven to be successful as reagents for targeted genome manipulation in Drosophila melanogaster and many other organisms. Their utility has been limited, however, by the significant failure rate of new designs, reflecting the complexity of DNA recognition by zinc fingers.
Kelly J. Beumer+8 more
openalex +5 more sources
Targeted Editing of Myostatin Gene in Sheep by Transcription Activator-like Effector Nucleases [PDF]
Myostatin (MSTN) is a secreted growth factor expressed in skeletal muscle and adipose tissue that negatively regulates skeletal muscle mass. Gene knockout of MSTN can result in increasing muscle mass in sheep. The objectives were to investigate whether myostatin gene can be edited in sheep by transcription activator-like effector nucleases (TALENs) in ...
Xinxia Zhao+9 more
openalex +5 more sources
No excessive mutations in transcription activator-like effector nuclease-mediated α-1,3-galactosyltransferase knockout Yucatan miniature pigs [PDF]
Objective Specific genomic sites can be recognized and permanently modified by genome editing. The discovery of endonucleases has advanced genome editing in pigs, attenuating xenograft rejection and cross-species disease transmission. However, off-target
Kimyung Choi+3 more
doaj +2 more sources
Rh D blood group conversion using transcription activator-like effector nucleases [PDF]
AbstractGroup O D-negative blood cells are universal donors in transfusion medicine and methods for converting other blood groups into this universal donor group have been researched. However, conversion of D-positive cells into D-negative is yet to be achieved, although conversion of group A or B cells into O cells has been reported.
Younghoon Kim+6 more
openalex +5 more sources
Transcription Activator-Like Effector Nucleases (TALEN)-Mediated Targeted DNA Insertion in Potato Plants [PDF]
Targeted DNA integration into known locations in the genome has potential advantages over the random insertional events typically achieved using conventional means of genetic modification. Specifically integrated transgenes are guaranteed to co-segregate, and expression level is more predictable, which makes downstream characterization and line ...
Adrienne Forsyth+3 more
openalex +5 more sources