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.
Not exact matches
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.
Alnylam has been developing drugs that
target gene expression in order to prevent to production of certain biological material
which can cause devastating diseases.
«Those carrying the
target gene variant slept, on average, for five hours,
which was one hour shorter than their twins without the
gene.
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.
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.
That could eventually lead to new drugs that selectively
target the
gene,
which McCarthy says, «could be a strategy for treating mood or sleep disorders.»
«Until recently we have been looking for individual
genes associated with diseases,
which drug companies then
target with treatments.
«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.
In particular, he has focused on
targeting the
gene coding for flagellin protein b, or flaB,
which has proved to be quite distinct from one genospecies to the next.
Women with precancer or cancer were most likely to have particular versions of the viral
gene E7,
which researchers are now interested in
targeting.
The Bill and Melinda Gates Foundation sees so much promise in
gene drive technology that it plans to double spending on its
Target Malaria initiative,
which aims to create systems for driving
genes in two species of malaria mosquitoes, to $ 70 million.
«This structural «footprint» we found seems to help these viruses get efficiently into the brain,
which informs the design of potentially safer brain -
targeted gene therapies,» said study senior author Aravind Asokan, PhD, associate professor of genetics.
The scientists injected the USH1G
gene into the inner ear using the innocuous AAV8 virus,
which enabled them to specifically
target the hair cells.
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.
Homozygous mutant mice in
which the Jak3
gene had been disrupted were generated by
gene targeting.
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.
Yang said the study not only indicated
which genes are affected by traumatic brain injury and linked to serious disease, but also might point to the
genes that govern metabolism, cell communication and inflammation —
which might make them the best
targets for new treatments for brain disorders.
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.
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.
«By greatly narrowing the specific
genes involved in social disorders, our findings will help uncover
targets for treatment and provide measures by
which these and other treatments are successful in alleviating the desperation of autism, anxiety and other disorders,» says Korenberg.
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.
Crispr, the programmable part, is the mechanism by
which bacteria identify and
target foreign
genes introduced by viruses.
«We captured about 95 percent of the
gene targets we set out to capture, many of
which were very large in DNA length,
which has been challenging in the past,» Parekkadan said.
The proposed strategy reflects a continuing move toward combination therapies, particularly when it comes to epigenetic approaches,
which target the mechanisms that control whether
genes are switched «on» or «off.»
They achieved this by transiently disturbing interactions between
target genes and PcG proteins,
which are complexes involved in the repression of several
genes governing development.
The
target fragment binds to a
gene switch in the DNA,
which triggers the production of a colourful substance such as the protein that gives jellyfish a green glow under ultraviolet light, or proteins from bacteria that produce colour changes visible to the naked eye.
First described in the 1990s — a discovery that led to the 2006 Nobel Prize — RNAi is a process by
which organisms suppress the expression of
target genes through the action of small RNA segments that bind to corresponding
gene sequences.
If cells were infected with an influenza A mutant lacking NS1, they proceeded to produce large number of the molecular complexes required for RNAi,
which include a protein called Argonaute that slices through the
target gene.
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.
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).
Johns Hopkins researchers say they have identified a set of
genes that appear to predict
which tumors can evade detection by the body's immune system, a step that may enable them to eventually
target only the patients most likely to respond best to a new class of treatment.
For this study, Dr. Lou and his colleagues focused on triple - negative breast cancer,
which is difficult to treat, because it does not exhibit receptors for estrogen, progesterone or the HER - 2 / neu
gene,
which are
targets for many current breast cancer treatments.
«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.
SU101
targets the signal transduction activated by a messenger called platelet - derived growth factor,
which controls the
genes involved in tissue formation and wound healing.
«We are now working to identify the best initial medical
targets related to the Werner
gene,» Galas said to the press,
which was true.
In this research, the group looked at two variants of miR - 21, a microRNA «oncomiR» known to
target tumor suppressor
genes and
which is highly expressed in a number of cancers as well as other proliferative diseases such as psoriasis.
The researchers say the findings could play an important role in identifying
which mutated
genes, and
which types of cancer, could be
targeted to take advantage of the deficiency and ultimately help in treating the cancer.
If we know
which genes are responsible for breast cancer, then we can tailor therapies to
target those
genes specifically.»
The analysis suggests that patients with complex indels in KIT would benefit from drugs such as imatinib, sunitnib and sorafenib,
which target mutations in this
gene.
The Emory team recently found that at least three
genes modify the bacteria's protein - making factory, the ribosome —
which is also streptomycin's
target.
Male leopard lacewings sport flashy orange designs,
which may signal to females that for such a conspicuous
target to still be alive, he must have good
genes.
These findings prompted the researchers to explore whether DNA methylation status of PPARα
target genes,
which is modulated and established in a PPARα - dependent manner in early life, persists into adulthood.
This understanding of cancer's evolution has key implications for treatment, he noted, «In addition to pointing out that we will need to
target driver
genes that are mutated early in cancer, this evolutionary approach provides a method that accurately characterizes
which genes are being mutated early and late.
Ten of those
genes code for odor receptors,
which researchers anticipate could become
targets for future louse repellents.
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.
«The good news is that this finding predicts that patients missing either
gene should be sensitive to new therapies
targeting focal adhesion enzymes,
which are currently being tested in early - stage clinical trials,» says Shaw, who is also a member of the Moores Cancer Center and an adjunct professor at the University of California, San Diego.
It appears to do this by
targeting little knots in their DNA, called quadruplexes,
which are very different from normal DNA and
which are especially found in faulty
genes.
CRISPRs are bacterial immune systems that contain many defense enzymes such as the Cas9 «molecular scissors,»
which scientists including Hsu have engineered as a powerful DNA -
targeting gene - editing tool.