Not exact matches
In our tests, the
Gene Knockout Kit gave us greater
than 80 percent knock - out rates for seven
targets,» shares Shondra Miller, Ph.D., Director, Center for Advanced Genome Engineering at St. Jude Children's Research Hospital, on Synthego's web site.
«Those carrying the
target gene variant slept, on average, for five hours, which was one hour shorter
than their twins without the
gene.
Since it is proteins that do the reprogramming, rather
than the
genes that make them, the researchers reasoned that they simply needed to get enough of the proteins into the
target cells.
«The state of the art right now is
targeting two or three
genes simultaneously and then looking at the effects, but we think that perhaps the
gene sets that need to be modulated to address some of these diseases are actually broader
than that,» says Lu, who is the senior author of the study.
Parkinson contends that
targeted gene replacement produces cells having specific genetic alterations far more effectively
than the traditional technique for making transgenic animals, which entails injecting DNA into cell nuclei.
It is typically less expensive to get preselected information about the 20,000 or so
genes that make up a person's exome — the section of the genome that provides instructions for making proteins —
than to perform a more precision - oriented test that
targets a single
gene.
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).
«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.
The same process has been studied as a potential genetic therapy for more
than a decade, because you can
target any disease
gene with matching dsRNA.
Rather
than target a tumor - suppressor
gene directly, Ideker and team took the approach of identifying genetic interactions between a tumor suppressor
gene and another
gene, such that simultaneous disruption of both
genes selectively kills cancer cells.
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.
«ALK now becomes the second abnormal
gene that we are able to successfully
target in lung cancer with drugs other
than chemotherapy.»
Beginning in 2012, researchers learned to modify this mechanism to precisely
target unwanted
genes rather
than viruses.
Because the vaccine causes the body to mount an immune response directed against a unique tumor, the therapy is much more effective
than gene -
targeted or more general chemotherapy alone.
It includes more
than 14,000 drug -
gene interactions involving 2,600
genes and 6,300 drugs that
target those
genes.
Importantly, mutations in the BRAF
gene that are seen in more
than half of advanced melanomas were absent in mucosal melanoma, explaining the ineffectiveness of BRAF -
targeted treatments like vemurafenib.
In fact, the researchers point out that phase 1 clinical trials are already underway for compounds
targeting this
gene in other cancers, meaning that the time needed to apply a similar strategy to mucosal melanoma could be dramatically shorter
than if they had to start from scratch.
Given his training in developmental biology, Raman focused the team to seek a novel drug
target on
genes important to the development of model organisms — fruit flies (Drosophila) and yeast (Saccharomyces cerevisiae)-- rather
than on oncogenes that transform a normal cell into a cancer cell.
One of the
gene areas identified suggests that cells within the lining of the uterus play a larger -
than - expected role in the length of pregnancy, which in turn provides a new
target for medications to help prevent preterm birth.
More
than 20 of the overexpressed
genes can be
targeted with existing drugs to muffle them, suggesting patients could be tested and matched to these drugs, says TCGA investigator Douglas Levine of Memorial Sloan - Kettering Cancer Center in New York City.
Since these proteins serve as «master regulators» for controlling whole
gene programs, drugs against these
targets could have broader effects
than traditional ones that block single enzymes.»
from the University of Washington reported a surprising finding - when delivered via AAV infection,
gene targeting vectors with relatively short (1 kb) homology arms underwent homologous recombination at a frequency three orders of magnitude higher
than identical
targeting vectors delivered through transfection of naked DNA
«It is therefore understandable that natural selection may have favored the relative
targeting of MMR to
genes rather
than non-genic regions.
In that year, Russell and Hirata from the University of Washington reported a surprising finding - when delivered via AAV infection,
gene targeting vectors with relatively short (1 kb) homology arms underwent homologous recombination at a frequency three orders of magnitude higher
than identical
targeting vectors delivered through transfection of naked DNA.
We are investigating the
genes that are the
target of this pathway, how it influences the cells of growing worms, the size differences between the sexes (hermaphrodite worms are larger
than males), and the way in which it mediates signals from the environment.
The study also showed that WGS identified more clinically actionable mutations
than the current standard of examining a limited subset of
genes, known as a
targeted panel.
Dr. Richard Pazdur, cancer drugs chief at the Food and Drug Administration, warned that although everyone hopes that
targeting drugs to
gene mutations will improve survival, «this may be far more complex
than we realize.»
Homozygous lethality of the XPCS allele is likely due to reduced levels of expression of this essential protein as a result of
gene targeting (Figure 1A) rather
than to the mutation itself.
What these groups are attempting basically is «
gene silencing», with a twist - it's aimed directly at the DNA, rather
than targeting a «message molecule» called RNA.
So, a drug that
targets CAG tracts could switch off those
genes too, which could end up doing more harm
than good.
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.
PrediXcan has the potential to identify
gene targets for therapeutic applications faster and with greater accuracy
than traditional methods.
The
gene produces an enzyme (diacylglycerol kinase eta) that functions at a point closer to the root of the lithium - sensitive pathway
than does the protein that lithium is thought to
target.
The majority of
gene -
targeting experiments in mice are performed in 129Sv - derived embryonic stem (ES) cell lines, which are generally considered to be more reliable at colonizing the germ line
than ES cells derived from other strains.
The CRISPR / Cas technology applied to mouse genetic engineering could quickly advance scientific understanding of disease mechanisms by allowing researchers to ask complex questions and find answers much faster
than with traditional
gene targeting approaches.
Given the tailored inhibition of selected
genes and the added precision brought by
targeted delivery systems, RNAi - based therapies are thought to carry lower risk of failure
than traditional approaches as the biological effects are more predictable.
They discovered that CRISPRi was much more efficient
than CRISPR - Cas9: in more
than 95 % of the cells created using CRISPRi, the
target gene was silenced, compared with only 60 - 70 % of cells grown from CRISPR - Cas9.
But rather
than changes in doublesex itself, these studies revealed changes in downstream
targets of dsx, via changes to specific DNA sequences to which DSX protein binds in the cis - regulatory regions of the bric - a-brac and desatF
genes and affecting sex differences in abdominal pigmentation and pheromone production.
In research that began more
than a decade ago, Biswal found GAUT4 expressed in poplar and then
targeted the
gene in both poplar and switchgrass.
Comparing this approach to the classic CRISPR / Cas9 system designed to cleave the DNA, the Gladstone team found that CRISPRi is actually more effective, silencing the
target gene in more
than 95 percent of cells, compared with the 60 percent to 70 percent efficiency of CRISPR / Cas9.
A variation of the
gene - editing technique can more precisely and efficiently downregulate the expression of
target genes than traditional CRISPR / Cas9.
Prolonged in vitro culture during
gene targeting found D4 to be more susceptible to differentiation
than C2.
CRISPR is now so well known that Google finally stopped suggesting I may be looking for «crisps» instead, but the real - world applications are not so well worked out yet, and there are various issues around CRISPR, including off -
target effects, and also the fact that deleting
genes is much easier
than replacing them with something else.
A subset of these miRNA knockout lines are «cluster» knockouts in which more
than one
gene has been
targeted.
To date, only 100 or so of the more
than 20,000
genes in the human genome have been developed as
targets for FDA - approved drugs.
It is always possible that a vector will introduce the
gene into a cell other
than that for which it is supposed to be
targeted (e.g., a spermatocytic cell) or that through a secondary mechanism
target cells that have taken up the new
gene will through some independent natural process (such as transfection) transfer the
gene to a germline cell.