Sentences with phrase «used gene targeting»

We used gene targeting to knock out both copies of the p21 (CIP1 / WAF1) gene in presenescent human fibroblasts.

Not exact matches

Gene therapy, for example, frequently involves using a virus to deliver a gene to the cells that scientists are targetGene therapy, for example, frequently involves using a virus to deliver a gene to the cells that scientists are targetgene to the cells that scientists are targeting.

That's not to say gene editing is new (it isn't), but Crispr simplifies the process by using molecular scissors that can be precisely targeted to snip out aberrant regions of genetic code, which can then be replaced with the correct sequences.

Well, one startup is seeking to take a very different approach: Exonics Therapeutics, which has secured $ 5 million in seed funding from CureDuchenne Ventures to see if the revolutionary new CRISPR - Cas - 9 gene - editing technology can be used to target the root genetic deficiency at the heart of the disease.

Using a gene - sequencing technique known as whole - exome sequencing that targets only the DNA that encodes proteins, the researchers cataloged virtually every significant DNA variant in three families in which the condition is common.

In addition, they also used exome sequencing and RNAseq — sequencing directly targeted to the SHIP1 gene — to examine the structure of the SHIP1 gene at both the DNA and the RNA level.

The vaccine can be targeted to the chosen pathogen by using RNA molecules which share sequence identity with the pest's genes and prevents their expression.

«The extensive overlap in risk genes for autism and cancer, many of which are chromatin remodeling factors, supports the idea of repurposing epigenetic drugs used in cancer treatment as targeted treatments for autism,» said Yan.

New research at the University at Buffalo reveals the first evidence that it may be possible to use a single compound to alleviate the behavioral symptoms by targeting sets of genes involved in the disease.

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.

Ko is working to adapt a procedure used so far in pigs, cows and other animals to target genes in cloned dogs.

«Many diseases, especially complex diseases, involve multiple genes, and this system could be used therapeutically to target and activate multiple genes together and rescue these disease phenotypes,» says Albert Cheng, a graduate student in the Jaenisch lab and co-author of the Cell Research paper.

«We spent half a dozen years trying to target the beta globin gene using the old technology,» he noted, adding that within one week of trying CRISPR, they had an editing tool that worked much better.

By using engineered zinc - finger nucleases (ZFNs) designed to target an integrated reporter and two endogenous rat genes, Immunoglobulin M (IgM) and Rab38, we demonstrate that a single injection of DNA or messenger RNA encoding ZFNs into the one - cell rat embryo leads to a high frequency of animals carrying 25 to 100 % disruption at the target locus.

Using this process, scientists can make targeted mutations in the genomes of living animals, either deleting genes or inserting new ones.

The scientists injected the USH1G gene into the inner ear using the innocuous AAV8 virus, which enabled them to specifically target the hair cells.

The finding suggests that other AAVs used for a gene therapy targeting the brain or spinal cord might be improved by having the same or a similar set of amino acids.

It is a type of branched DNA ISH that uses pairs of «Z - probes,» which are highly specific to target genes, but also small enough to easily diffuse into the tissues under study.

«Neuroscientists are using these new gene - editing and molecular tools to develop potential therapeutic targets across multiple disease fronts.»

Dr. Satish Rattan, Professor in the Department of Medicine, Division of Gastroenterology and Hepatology at Jefferson's Sidney Kimmel Medical College, together with Drs. Jagmohan Singh and Ipsita Mohanty, used altered copies of the body's own genetic make - up — small RNA fragments (microRNAs) that regulate the target gene RhoA / ROCK — in order to strengthen or weaken the muscle tone of the sphincter.

Not least the fact that gene targeting using homologous recombination or any other method is not perfect — the added DNA is sometimes inserted into the wrong place in the genome, and the process can trigger other kinds of mutations too.

However, they determined the blood levels of SMN are not altered when SMN levels are altered in motor neurons using a gene therapy approach delivered directly into the cerebrospinal fluid to target the central nervous system.

A carrier called a vector must be used to deliver the therapeutic gene to the patient's target cells.

Theoretically, it could be simpler and more targeted to use gene blueprints from sequencing data to build something that mimics microRNAs — binding to the same things the microRNA would — then embarking on a fecal transplant and ensuring it is safe.

The researchers found a set of frequently mutated genes in SBAs that could be helpful to clinicians when they are looking to use targeted therapies that work best in cancers with specifics mutations.

«By using chloroplast transformation we generated potato plants that accumulate high amounts of long stable dsRNAs targeting essential genes in the beetle,» says Ralph Bock.

Identification of the genes represents a vital step toward developing compounds that can be used in targeted, individualized treatment of severe periodontitis, before loss of teeth and supportive bone occurs.

The former target, say, using gene editing techniques to inactivate HIV receptors and achieve resistance of blood cells to the virus (which Sangamo BioSciences is working on in clincial trials) is different than helping parents who both carry genes for Huntington's Disease to have a child that is free of the disease (a change to the genome that would be passed on to future generations and would likely not be very commonly needed).

By using gene drive, a method targeting specific genes, they could go in and tip the scale in the mosquito's favor.

«The genes which these miRNAs regulate also had increased levels, indicating that these gene expression, indicating that these gene products were likely targeted for storage and for possible future use within the brain cell, rather than for destruction.

Furthermore the study by Huang et al showed that the although the CRISPR / Cas9 technique they used can work in the embryo, it can miss the target in the gene and is too inefficient.

Scientists have developed a three - in - one blood test that could transform treatment of advanced prostate cancer through use of precision drugs designed to target mutations in the BRCA genes.

«Depending on the research questions, it may also be important to define the appropriate strategy to use, either whole - genome sequencing or just targeting some genomic regions with a gene baiting approach,» says Besnard.

The researchers found that gene drive is unlikely to work for most mosquito genes because they are too variable in nature, however they also used the data to highlight less variable targets that are potentially more suitable for gene drive based methods to control mosquitoes.

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.

In gene - baiting, scientists use targeted PCR primers to amplify specific genes during the library preparation step, rather than amplifying all of the DNA fragments in the sample.

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.

For instance, researchers used virtual screening to identify compounds that target the gene for PFKFB3, an enzyme that helps regulate the metabolism of cancer cells.

They tested their system on a pair of yeast transcription factors and used the data to predict which yeast genes the proteins would target, they report in this week's Science.

Still, the standard form of liver - targeted gene therapy carries a range of potential complications, including the risk of harmful mutations and of the body mounting an immune response against the viral vectors used to carry the correct forms of the defective genes responsible for haemophilia.

The authors also provide experimental validation for using active genetics as an efficient means for targeted gene insertion, or «transgenesis,» and single - step replacement of genetic control elements.

«In the current study, using a genome - wide analysis of DNA methylation, we identified a few PPARα target genes that underwent ligand - activated PPARα - dependent DNA demethylation during the perinatal period and whose DNA methylation status persists into adulthood,» explains a corresponding author Koshi Hashimoto.

Using a gene - editing system that can disable any target gene, they have shown that they can selectively kill bacteria carrying harmful genes that confer antibiotic resistance or cause disease.

«So, with this knowledge, the dream is that we could eventually use gene editing technology to edit the microRNA target sites in the host plants, preventing the microRNAs from binding and silencing these genes.

Dr. Jones obtained his Ph.D. in 2003 from the University of Birmingham Institute of Cancer and Genomic Sciences (United Kingdom) under Professor Lawrence Young, studying the use of gene therapy for targeting Epstein - Barr Virus (EBV) proteins with replication - competent adenoviruses to treat EBV - driven malignancies.

As the central nervous system is also a target of AAV vectors, Hélène Puccio and Patrick Aubourg's teams are investigating whether a similar approach using gene therapy could be as effective for the spinal cord and cerebellum as it is for the heart.

The audience will learn about the current state of breast cancer research, how data generated by NGS gene panels target variants of interest and have been developed and used in routine laboratory research, and the broader issues of breast cancer education, awareness, and community services.

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.

He has worked in the biotech industry as a research scientist for over 11 years with a focus on emerging technologies including gene targeting in mice, molecular analysis of transgenes using GFP variants at the single cell level, and developing flow cytometry reagent kits to speed up assay development time for researchers.

Drugs used in targeted gene therapy operate by inhibiting one or more cellular processes.