Their research has created the technology known
as gene targeting, now used in virtually all areas of biomedicine — from basic research to the development of new therapies.
Competitive pathway analysis implicated the biological processes of neurogenesis and synaptic regulation, as well
as the gene targets of two pharmacologic agents: cinnarizine, a T - type calcium channel blocker, and LY97241, a potassium channel inhibitor.
Not exact matches
Luxturna is the first of a crop of treatments that
target diseases caused by mutations in specific
genes, and thus is referred to by many
as the first
gene therapy in the U.S.
The Kamens claim the main thing that distinguishes their foundation from other brain tumor foundations is their focus on pediatric brain cancer specifically,
as well
as their close ties with pharmaceutical and biotech companies working in the fields of immunotherapy and
target gene therapy.
During the dialogue, Dr
Gene Nisperos of the Health Alliance for Democracy raised his concern
as the
target of Golden Rice are members of the vulnerable population such
as preschool children and lactating mothers.
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 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.
As the
target gene's methylation changes, so does the reporter's.
The study, published online today in Science Translational Medicine, further points to a master switch for these
gene sets
as a potential
target of future therapies.
In all the pathways so far investigated, the final result is a gradient of morphogen that functions principally
as a transcription factor, initiating or suppressing the transcription of one or more
target genes in a concentration - dependent manner.
Recent advances in the understanding of cancer have led to more personalized therapies, such
as drugs that
target particular proteins and tests that analyze
gene expression patterns in tumors to predict a patient's response to therapy.
They note that
targeting inflammation to treat infections offers an advantage over antibiotic therapy,
as the former hinders
gene transfer and the evolution of pathogens, while the latter promotes bacterial evolution and, ultimately, antibiotic resistance.
If so, the master
genes would be even more promising
as targets for new treatments.
Earlier MD Anderson investigations demonstrated exosomes
as a factor in detecting pancreatic cancer, but these latest findings reveal genetically altered exosomes
as a potentially novel approach for direct and specific
targeting of mutated KRAS, the cancer
gene commonly linked to pancreatic cancer.
The team had expected to find changes in many
genes involved in serotonin signaling, a process
targeted by some popular antidepressants such
as Prozac.
Mutations to these
genes can affect the choice of therapy
as well
as how to better
target the mutations.
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.
The researchers are now investigating the effects of these
genes» mutations
as possible
targets for new therapeutic treatments.
Starting with a candidate list of 20,000
genes, researchers screened down to eight before singling out VNN - 1
as the primary
target.
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.
The
gene expression similarities between this common condition and the current findings in late - stage RP suggest that the canine RP models could serve
as stand - ins to evaluate the effectiveness of pharmaceutical interventions that
target innate immunity pathways in AMD
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.
«Such changes may affect other
genes as well; we haven't studied all the potential
targets yet.
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.
NIPD for single
gene disorders in a fetus is diagnostic,
as it
targets specific genetic changes present in a high risk family.
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.
Despite this high profile, K - Ras has earned a reputation in scientific circles
as being «undruggable» because many pharmaceutical, biotech, and academic laboratories have failed to design a drug that successfully
targets the mutant
gene.
Importantly, this monoubiquitination serves
as an integral part of SETDB1 to render its activity and leads to inhibition of
target gene expression.
But hopes that ageing could be delayed by
targeting a single
gene or protein in a single molecular pathway have faded,
as researchers have learned that the key pathways vary according to the animal.
«
Gene defect
as a potential gateway for
targeted prostate cancer therapy.»
By searching for
gene deletion patterns in cancer through a concept the investigators call «synthetic essentiality,» the team identified a synthetic essential
gene known
as chromatin helicase DNA - binding factor (CHD1)
as a therapeutic
target for prostate and breast cancers lacking a tumor suppressor
gene called phosphatase and tensin homolog (PTEN).
Further investigation uncovered CHD1's role
as vital to PTEN signaling, and
as a potential therapeutic
target in prostate and breast cancers with PTEN
gene loss.
«A retrospective analysis of the CHD1
gene in these samples may reveal the potential utility of CHD1
as a biomarker for improved prostate cancer patient stratification and
targeted therapy with PARP inhibitors,» notes Johnsen.
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.
Significant binding by miR -124-3p to two of the potential
target genes was seen,
as measured from immunoprecipitated RNA - induced silencing complexes.
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 hear
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 hear
as effective for the spinal cord and cerebellum
as it is for the hear
as it is for the heart.
They combined the protein with synthetic molecules, known
as PNAs, that mimic DNA and bind to the
target gene to form a triple helix.
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.
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.
But only after engaging the
target and adjusting their antigen receptors over many generations (
as B cells do by mutating their antigen receptor
genes) can this lineage of cells produce VRC26 bnAbs.
Experimental approaches such
as gene therapy are also being investigated, but Dr. Rudnicki's research suggests that these approaches will have to be modified so that they
target muscle stem cells
as well
as muscle fibres.
«Since this
gene has previously been identified
as a
target for the development of new drugs, in the future, it may be possible that early detection will facilitate better management of this disease.»
By mapping the neural processes involved
as birds learn mating songs, scientists hope to someday use that knowledge to
target specific
genes disrupting speech in patients with autism or other neurodevelopmental conditions.
Many cancer drugs
target one of the soldier proteins that have already been expressed from a
gene, have received marching orders from other proteins, and are well on their way to causing problems, such
as metastasis.
Professor Moffatt said: «The
genes we identified represent new potential drug
targets for allergic diseases
as well
as biomarkers that may predict which patients will respond to existing expensive therapies.»
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.
In animals such
as mice, it is fairly routine to study
gene functions by deleting a
target gene or replacing it with an artificial piece of DNA.
Led by Massey's Deputy Director Steven Grossman, M.D., Ph.D., a team of scientists
targeted the
gene CtBP with a drug known
as HIPP (2 - hydroxy - imino phenylpyruvic acid) and were able to reduce the development of pre-cancerous polyps by half and return a normal lifespan to mice born with a predisposition to intestinal polyps.
The non-pathogenic vectors serve
as gene shuttles to transport components of SIV, such
as envelope proteins, into the
target cells.
The first group was mice with
targeted deletion of the interleukin 10 (IL10)
gene, known
as interleukin 10 knockout (IL10 - / --RRB- mice, which develop colitis through T helper immune cells.