These «genetic scissors» can be used for
targeting any gene in a cell in order to modify it.
In their July 2014 paper, Esvelt and his colleagues described how gene drive would work: CRISPR would cut
a target gene in a cell or cells, probably an egg or embryo.
Not exact matches
The technology's possibilities are staggering —
in theory, allowing medical scientists to do everything from cure genetic disorders like sickle
cell disease to identify
gene targets for combating HIV.
I won't reveal yet who my favorites are, but I will say that these young scientist - founders came up with very creative solutions for preventing infections
in some common surgeries, tackling resistance
in targeted antibody drugs, improving
gene vectors for
cell therapies, helping the vision - impaired «see» faces and better read their environments, imaging hard - to - see spots
in the lungs and other organs, improving genetic risk analysis, and expediting the logistical operations of hospitals.
To discover these
targets, the team determined when and where each
gene is turned on or off
in the
cells and tissues of H. contortus to reveal new insights into the worm's lifecycle.
Because the
genes that are massively amplified
in the neochromosomes are essential for the
cell's survival, drugs that turn those off are an obvious
target for research.
In SIF - seq, hundreds or thousands of DNA fragments to be tested for enhancer activity are coupled to a reporter gene and targeted into a single, reproducible site in embryonic cell genome
In SIF - seq, hundreds or thousands of DNA fragments to be tested for enhancer activity are coupled to a reporter
gene and
targeted into a single, reproducible site
in embryonic cell genome
in embryonic
cell genomes.
«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.
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.
The findings by a team of Massachusetts General Hospital (MGH) investigators, which will be published
in the April 24 issue of
Cell and are receiving advance online release, support the importance of epigenetics — processes controlling whether or not
genes are expressed —
in cancer pathology and identify molecular circuits that may be
targeted by new therapeutic approaches.
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.
Gene Yeo, a professor of cellular and molecular medicine at UCSD, led the research and showed he could
target RNA
in living
cells, a first step toward treating diseases like muscular dystrophy and neurodegeneration.
This
cell's
gene - editing system
targets RNA, revealing the molecule's distribution
in the cytoplasm.
Gene switches have been identified that work
in specific brain areas, potentially enabling
targeted treatment of unhealthy
cells.
In this way, the team identified a new small protein, growth inhibitor
gene product (Gp) 0.6, which specifically
targets and inhibits the activity of a protein essential to bacterial
cells.
Dwarki and Jaime Escobedo improved the AAV's ability to insert
genes into chromosomes by adding a
gene promoter region from cytomegalovirus, known to be active
in the
target for their
gene therapy, muscle
cells.
In a two - pronged attack, these viruses specifically
target tumor
cells while delivering a cargo of immune - boosting
genes.
CTL119 manufacturing begins with a patient's own T
cells, some of which are removed and then reprogrammed
in Penn's Clinical
Cell and Vaccine Production Facility with a
gene transfer technique designed to teach the T
cells to
target and kill tumor
cells.
«Margery's experiments showed that the NS1 protein can alter expression of Hedgehog
target genes on its own, without other viral proteins,» said Bier, professor and newly named holder of the Tata Chancellor's Endowed Professorship
in Cell and Developmental Biology.
Bach2, an important
gene for inducing memory B
cells, may become an important
target in vaccine strategies.
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).
These receptors, called receptor tyrosine kinases (RTKs), transmit instructions through the
cell wall and down through a cascade of reactions to a
target gene in the nucleus.
With high reproducibility, the sgRNAs designed with CrispRGold destroyed the
target genes in on average 80 percent of the
cells — «an excellent rate,» says Graf.
A molecule
in cells that shuts down the expression of
genes might be a promising
target for new drugs designed to treat the most frequent and lethal form of brain cancer, according to a new study by researchers at The Ohio State University Comprehensive Cancer Center — Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC — James).
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 an effort to expand the number of cancer
gene mutations that can be specifically
targeted with personalized therapies, researchers at University of California San Diego School of Medicine and Moores Cancer Center looked for combinations of mutated
genes and drugs that together kill cancer
cells.
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 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 dru
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 dru
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 dru
in groups of
cells to identify those involved
in resistance to a melanoma dru
in resistance to a melanoma drug.
When Cas9 and the short guide RNA
targeting a disease
gene are delivered into
cells, a specific cut is made
in the genome, and the
cells» DNA repair processes glue the cut back together, often deleting a small portion of the genome.
«Research into basic workings of immune system points to way of improving therapies for cancer: Differences
in wiring of «exhausted» and effective T
cells indicate possible
gene - editing
targets.»
«Identifying
targets essential to
cell survival
in tumor suppressor
genes has long been an investigational goal with the aim of offering cancer - specific vulnerabilities for
targeted therapy,» said Ronald DePinho, M.D., professor of Cancer Biology, MD Anderson president, and senior author for the Nature paper.
His team's approach is based on
gene therapy, where a «tame» virus is harnessed to transfer a
gene into
target cells in the recipient.
One potential treatment for CF is
gene therapy, and a major challenge
in gene therapy is packaging replacement
genes so they can be delivered to the
target cells.
But why does WOX5 switch off its
target gene CDF4
in stem
cells?
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.
In 2013, CRISPR passed two important tests: It works in human cells, and it can target several genes at onc
In 2013, CRISPR passed two important tests: It works
in human cells, and it can target several genes at onc
in human
cells, and it can
target several
genes at once.
«Heart toxin reveals new insights into HIV - 1 integration
in T
cell genome: Digoxin exposes link between T
cell activation and
targeted HIV - 1 integration
in specific
genes.»
In both human airway cells and mouse nasal cells, the researchers observed corrections in the targeted gene
In both human airway
cells and mouse nasal
cells, the researchers observed corrections
in the targeted gene
in the
targeted genes.
If so, it could make
cell fate more resilient to random mutations
in a plant's genetic code, even when such changes keep some
gene - regulating proteins from binding their intended DNA
targets.
Intrigued by the ability of certain polymers to mop up DNA and RNA for
gene transfer, Sullenger and colleagues tested the idea that these chemical compounds might also be effective
targeting such nucleic acids as they arise
in cell death.
Gene switches have been identified that work
in very specific brain areas, potentially enabling light to
target unhealthy
cells without disrupting healthy ones.
These include Cenix Bioscience, which has developed a new lab technique to identify the
genes that are involved
in cell division, predicted to be very useful for finding potential new
targets for anticancer drugs.
Research fields are diverse, including basic research on
cell proliferation, analysis of tumour
cells and tissues to detect
gene mutations, and identification of potential therapeutic
targets in cancer.
Furthermore, besides offering valuable insight into the function of this novel RNA type, the researchers also believe that the findings will open new avenues for novel treatments
in which
cell - specific enhancer sequences can be
targeted to alter
gene expression.
«New treatment
targets cancers with particular genetic signature: Mutations
in gene SETD2 make cancer
cells vulnerable to drug inhibiting the protein WEE1.»
By synthesizing a nanoparticle that releases its siRNA cargo only after it enters
targeted cells, Dr. Tariq M. Rana and colleagues showed
in mice that they could deliver drugs that silenced the
genes they wanted.
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.
But so far, silencing
genes using RNAi has not worked well
in mammalian
cells; although the approach has been successful
in mouse embryos, double - stranded RNA shuts down synthesis of all proteins, not just the
target gene,
in other types of mammalian
cells.
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.