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This introduction video shows how the choice of every vector element can modify the lentiviral vector's activity. 
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Researchers sequence the entire genome of the Clostridium autoethanogenum bacterium, which is used to sustainably produce fuel and chemicals from a range of raw materials, including gases derived from biomass and industrial wastes.
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Researchers sequence the entire genome of the Clostridium autoethanogenum bacterium, which is used to sustainably produce fuel and chemicals from a range of raw materials, including gases derived from biomass and industrial wastes.
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Scientists are lending Mother Nature a helping hand in fight against cancer and Alzheimer’s with the development of a new, more effective way to make amino acids. The new 'designer proteins' can be used to make more effective drugs with fewer side effects, they report.
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The researchers designed a decoy protein to interrupt the signaling pathway that triggers the breakaway of cancerous cells. Preliminary tests showed this strategy effective in mice models; infusion with this decoy protein greatly reduced metastasis in mice with aggressive breast and ovarian cancers.
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The authors provide an online tool for the design of highly active sgRNAs for any gene of interest. Indeed, they discovered sequence features that improved activity, including a further optimization of the protospacer-adjacent motif (PAM) of Streptococcus pyogenes Cas9. The results from 1,841 sgRNAs were used to construct a predictive model of sgRNA activity to improve sgRNA design for gene editing and genetic screens.
http://www.broadinstitute.org/rnai/public/analysis-tools/sgrna-design
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From BioPortfolio: Type I CRISPR-Cas systems require a target-searching Cascade complex and the Cas3 degradation machine to drive prokaryotic adaptation to alien nucleic acids. Ca...
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The authors used CRISPR/Cas9–mediated genome editing to correct the dystrophin gene (Dmd) mutation in the germline of mdx mice, a model for DMD, and then monitored muscle structure and function. Genome editing produced genetically mosaic animals containing 2 to 100% correction of the Dmd gene. The degree of muscle phenotypic rescue in mosaic mice exceeded the efficiency of gene correction, likely reflecting an advantage of the corrected cells and their contribution to regenerating muscle.
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Differential knockdown of TGF-beta ligands in a three-dimensional co-culture ...
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The authors' observations suggest a mechanism for a Protospacer Adjacent Motif (PAM)-dependent target DNA melting and RNA–DNA hybrid formation. Furthermore, this study establishes a framework for the rational engineering of Cas9 enzymes with novel PAM specificities.
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The authors enhanced the integration site-specificity of the phiC31 integrase-based vector using a sequence-specific DNA-binding protein containing Gal4 and LexA DNA-binding motifs. In human cells, they showed thanks to quantitative PCR that integration around the LexA operator was 26-fold higher than with the control.
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The authors demonstrated in vivo as well as ex vivo genome editing using adeno-associated virus (AAV)-, lentivirus-, or particle-mediated delivery of guide RNA in neurons, immune cells, and endothelial cells using Cre-dependent Cas9 knockin mouse.
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A new primate model has been developed to test treatments that might cure HIV/AIDS and suggests answers to questions raised by the 'Berlin patient,' the only human thought to have been cured so far.
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After a high-throughput screening, the researchers have identified phorbol 12-myristate 13-acetate (PMA) as a candidate to enhance lentiviral transduction. Indeed, , the percentage of GFP-positive CD34+ cells was increased from 7% in the absence of PMA to greater than 22% in the presence of 1 nM PMA. PMA did not affect colony formation of CD34+ cells or the expression of the hematopoietic markers CD34 and CD45.
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The authors use CRISPR-Cas technology to create antimicrobials whose spectrum of activity is chosen by design.
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The authors has developed a CRISPR/Cas9 nuclease specific for the bovine Nanog locus They showed that this genome editing tool can be used for homologous recombination targeting in bovine pluripotent cells is an efficient gene editing method that can be used to generate transgenic livestock in the future.
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PLOS ONE: an inclusive, peer-reviewed, open-access resource from the PUBLIC LIBRARY OF SCIENCE. Reports of well-performed scientific studies from all disciplines freely available to the whole world.
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Hydrodynamic injection of the piggyBac vectors into hemophilia A mice temporally treated with an immunosuppressant resulted in stable production of circulating FVIII for over 300 days without development of anti-FVIII antibodies. Furthermore, tail-clip assay revealed significant improvement of blood coagulation time in the treated mice.piggyBac transposon vectors can facilitate the long-term expression of therapeutic transgenes in vitro and in vivo. This novel gene transfer strategy should provide efficient delivery of FVIII.
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A modified version of the Clostridium novyi (C. noyvi-NT) bacterium can produce a strong and precisely targeted anti-tumor response in rats, dogs and now humans, according to a new report from Johns Hopkins Kimmel Cancer Center researchers.
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The authors describe a new method of cancer model generation using the CRISPR/Cas system delivered in vivo by hydrodynamic injection in wild-type mice. This strategy open a new way for rapid development of liver cancer models and functional genomics.
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An international, peer-reviewed genome sciences journal featuring outstanding original research that offers novel insights into the biology of all organisms
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The authors combined CRISPR/Cas9 technology with the piggyBac transposon to efficiently correct the HBB mutations in patient-derived iPSCs without leaving any residual footprint. No off-target effects were detected in the corrected iPSCs, and the cells retain full pluripotency and exhibit normal karyotypes. When differentiated into erythroblasts gene-corrected iPSCs restored expression of HBB compared to the parental iPSCs line.
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Video which explains the influence of promoter, integrase or envelope on the activity of lentiviral vectors. The goal is to design the best vector according to the experimental needs.
http://geg-tech.com/