Sentences with phrase «human gene targeting»

«Human gene targeting by viral vectors.»

Not exact matches

'' «At PMV Pharmaceuticals, we are targeting the most frequently mutated gene in human cancer (p53) to make an unprecedented impact on cancer patients» lives.

If Rudenko confirms Clark's PCR tests using additional gene targets, and if Clark or Rudenko manage to grow Borrelia cultures out of human and lone star tick samples, then even the fiercest skeptics will have to recognize that Southern Lyme strains threaten human health, and that Lyme - like illness deserves Lyme - like treatment.

Our data demonstrate the feasibility of targeted gene disruption in multiple rat strains within 4 months time, paving the way to a humanized monoclonal antibody platform and additional human disease models.

Carlo Croce, a cancer researcher at Ohio State University in Columbus, and his colleagues created a diagram of interacting miRNAs for normal body cells by connecting them according to which genes they target and the function of those genes, in a way similar to analyses of human social networks.

After moving to Berkeley, he arrived at a career crossroads in 1994, when Spyros Artavanis - Tsakonas, then at Yale, discovered and subsequently patented the human relative of the fruit fly gene notch, which plays a role in cell - to - cell interactions and could be an anti-cancer target.

«If these genes would have a role [in humans], it would be very exciting to try to [make] medicines that target some of these genes,» Moreno says.

In one experiment with human cells, a guide RNA should have led the Cas9 enzyme only to a gene on chromosome 2 (yellow bar), but it also directed the enzyme to many off - target sites (red) on several other chromosomes.

The vector then unloads its genetic material containing the therapeutic human gene into the target cell.

Before moving on to human trials, they will need to study all instances of «off - target» effects: Years before Crispr, the viruses employed to deliver DNA in gene therapy trials occasionally damaged the whole system, causing cancer.

It turns out that each virus that's been studied and associated with cancer — such as hepatitis B with liver cancer or human papilloma virus with cervical cancer — evolves characteristics that allow it to target those genes immediately upon infection.

Yet a virus made so weak that it can not get us sick often can not even last long enough to deliver the gene to its target, being destroyed by the human immune system before it arrives.

This and other evidence, say study authors Svante Pbo of the Max Planck Institute for Evolutionary Anthropology in Leipzig, Germany, and his colleagues, «strongly suggest that this gene has been the target of selection during recent human evolution.»

Although that marker, called IL21, had not previously been associated with autoimmune diseases, the gene that produces it sits right in the stretch of DNA known to make these mice vulnerable to diabetes, suggesting that IL21 might make a drug target, says Sarvetnick.Furthermore, by giving the animals a shot of dead bacteria — similar to an immunization in humans — when they were newborns, Sarvetnick and her colleagues prevented a surfeit of CD4 + and CD8 + cells.

In humans, variants of the IL - 15R - alpha gene have been found in world - class endurance competitors, suggesting a target for gene therapies aimed at boosting the ability to exercise longer.

Using in vitro, or test tube, experiments, the researchers applied these chemicals to human cancer cells to measure changes of estrogen receptor - and androgen receptor - target genes and transcriptional activity.

The researchers first used yeast to quickly and cheaply screen 169,000 interactions between yeast versions of human tumor - suppressor genes and genes that can be inhibited with drugs, sometimes called «druggable» targets.

In one experiment this year, a team led by another CRISPR pioneer, Feng Zhang of the Broad Institute in Cambridge, Massachusetts, targeted the 20,000 or so known human genes, turning them on one by one in groups of cells to identify those involved in resistance to a melanoma drug.

Researchers from KTH Royal Institute of Technology's Science for Life Laboratory (SciLifeLab) research center and Gothenburg University employed the biological networks generated for 46 major human tissues in order to identify the liver - specific gene targets.

Using a recently developed genome - editing technique called CRISPR, a Chinese team has successfully altered two target genes in cynomolgus monkeys, paving the way for the development of monkey models that mimic human diseases.

Ras is the most frequently mutated human cancer gene (oncogene), yet despite recent breakthroughs, therapeutic options to target Ras - dependent cancers remain limited.

In 2013, CRISPR passed two important tests: It works in human cells, and it can target several genes at once.

Previous research has shown that HIV - 1 integrates more frequently into human genes that are transcribed into RNA (the first step in gene expression), but the biological significance of this targeting has been unclear.

Derived mostly from human embryonic kidney 293T (HEK293T) and HeLa cell lines, EdiGene Knockout (KO) Cell Lysates have been optimized through the use of genome editing technology and validated at the genomic level through PCR and Sanger - sequencing techniques to ensure the accuracy and knockout of the target gene.

In both human airway cells and mouse nasal cells, the researchers observed corrections in the targeted genes.

Earlier versions of these «base editors,» which target typos related to the other half of disease - causing genetic spelling errors, have already been used to alter genes in plants, fish, mice and even human embryos.

In 2006 Pfizer started early human testing on one of these new, targeted drugs called crizotinib (now sold as Xalkori), concentrating on a mutation of a gene called MET, implicated in several cancers, including esophageal and stomach cancer.

Most simply, once these genes, or bits of DNA tied to the genes (known as markers), have been identified, molecular breeders can quickly target offspring inheriting the genes for further development, cutting breeding time and improving the crop's «genetic gain,» the generational improvements made to a crop, like increased height, by human selection.

Whereas in the nematode experiment the researchers targeted nanoparticles to temperature - sensitive ion channels that naturally exist in the membranes of the worms» nerve cells, the scientists inserted the gene for a heat - activated ion channel called TRPV1 into the human and rat cells.

He says HGS was getting «diminishing returns» from its investment in TIGR since Venter had steered his outfit into sequencing organisms of little medical importance, and into human genome sequencing, also of limited value for a company like HGS that is interested in genes as drug targets (not untranslated DNA that makes up most of the genome).

The next step was to find out which role Shp2 and its target genes play in human patients with breast cancer.

One ZFN pair was used to target both the human and rhesus macaque CXCR4 genes since the 24 bp target sequences are identical.

The team designed the PshRNA agent selectively targeting the common sequence of human and mouse (pro) renin receptor genes.

Asuri, Prashanth, et al. «Directed evolution of adeno - associated virus for enhanced gene delivery and gene targeting in human pluripotent stem cells.»

For gene editing to work, it is essential to uniquely target a single site among the 3 billion nucleotides in the haploid (single set of unpaired chromosomes) human genome.

The first successful targeted human gene therapy was reported in 2000.

Caribou's market - leading CRISPR - Cas9 gene editing technology can accurately target and cut DNA to produce precise and controllable changes to the genome, which can be applied by JAX to create mouse models that better recapitulate human diseases enabling researchers to find better treatments faster.

This was achieved after correction of the human sickle hemoglobin allele by gene - specific targeting.

Thus, neural derivatives of disease - specific human pluripotent stem cells constitute a relevant biological resource for exploring the impact of adult - onset HD mutations of the HTT gene on the division of neural progenitors, with potential applications in HD drug discovery targeting HTT - dynein - p150Glued complex interactions.

Although CRISPR is incredibly useful for generating mutations by NHEJ and generating small mutations with HDR, when it comes to larger scale genome editing, such as replacement of a mouse gene with its human ortholog (greater than 5 kb), it remains to be seen whether CRISPR is as robust as conventional gene targeting.

This is a great example of a potential gene therapy target that still needs a fair amount of work to validate the thesis and the initial data, but having a large number of existing human carriers is a good sign on the safety front.

Being able to go back and forth between the mouse and human genomes so easily has also made it much simpler and quicker to target related human genes that could be candidates for drug development.

NGF is in fact viewed as a viable target for AD clinical trials with one group investigating NGF ex vivo gene delivery in a Phase 1 trial with human patients aimed at stimulating cholinergic function and improving memory [123].

The sequences of small interfering RNA (siRNA) targeting the human mTOR, AKT1, and 4E - BP1 gene products were purchased from Ambion, Inc. (Austin, TX) and were as follows: mTOR sense GGAGUCUACUCGCUUCUAUTT and antisense AUAGAAGCGAGUAGACUCCTC, AKT1 sense GGGCACUUUCGGCAAGGUGTT and antisense CACCUUGCCGAAAGUGCCCTT, and 4E - BP1 sense GGUACCAGGAUCAUCUAUGTT and antisense CAUAGAUGAUCCUGGUACCTC.

We focus on developing computational methods and tools for (a) analyzing large - scale gene expression data related to human cancer in search for gene markers and disease sub-categories, (b) identifying regulatory elements such as miRNA precursors and their targets in whole genomes of plants and mammals, (c) building theoretical models of gene regulatory networks.

And CRISPR has completely and utterly transformed the field, as we can do rapid gene targeting now in primary human neural stem cells.»

We conclude that the large number of mouse mutants and human de novo mutations may be due to the combination of the Chd7 gene being a large target and the fact that many heterozygous carriers of the mutations are viable individuals with a readily detectable phenotype.

In MMEJ pathway, we achieve efficient gene disruption in human cell lines and animals by developing a computer program that assists the choice of nuclease target sites based on microhomology prediction.

Following a Forward Genetics approach, Fleming researchers identified a novel neurological mouse model caused by a functional mutation in the Slc25a46 gene, a new pathogenic target in a wide spectrum of human neurological diseases, including optic atrophy, Charcot - Marie - Tooth type 2, Leigh syndrome, progressive myoclonic ataxia and lethal congenital pontocerebellar hypoplasia.

Targeted Gene Addition to a Safe Harbor locus in human CD34 + Hematopoietic Stem Cells for Correction of X-linked Chronic Granulomatous Disease.