Sentences with phrase «for therapeutic gene»
These alternate strategies include using small - molecule drugs that affect RNA metabolism or protein stability, as well as administering modified viruses for therapeutic gene delivery (see «Getting a fix on SMA»).
http://www.smafoundation.org/ Not exact matches
The OAR proposal uses a variation of therapeutic cloning called altered nuclear transfer (ANT) in which the nucleus of a donor cell (a skin cell, for example), containing the 30,000 genes of the genetic code, is altered in such a way that it produces an epigenetic factor, a protein called nanog.
«The advantage of being able to adjust a set of genes identified as key autism risk factors may explain the strong and long - lasting efficacy of this therapeutic agent for autism.»
«My team at Nationwide Children's has worked with commitment and dedication to develop a therapy that may subsequently be shown through future clinical trials to potentially alter the course of this unforgiving condition and provide a therapeutic option for the families and infants with SMA1,» says Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at Nationwide Children's.
The problem, Sweeney says, is that the viruses researchers use for delivering therapeutic genes infect primates but not other mammals.
This fact is rooted in a packaging problem: the gene encoding FIX is much smaller than the gene for FVIII, and better fits into a vector, usually a harmless virus, designed to deliver therapeutic genes to a patient.
For gene therapies, scientists remove most of the AAV genome, replace it with therapeutic genetic cargo, and inject trillions of copies into the patient.
To use viruses as delivery vehicles for gene therapy, researchers take all the harmful and replicative genes out of the virus and put in the therapeutic genes they want to deliver.
«We showed that the deletion of the gene responsible for this pathway can restore the original therapeutic function of the cells,» said Prof. Leor.
«The number of protein functions that are currently targeted by drugs is incredibly small compared to the total number of protein interactions that could be targeted for therapeutic benefit,» says Geoffrey Wahl, a professor in Salk's Gene Expression Laboratory.
This work provides a proof of principle for a new approach in the study and treatment of cancer: the aberrant expression in a tissue or organ of genes specific to other tissues could become a new tool for establishing a prognosis and personalizing therapeutic care.
The researchers are now investigating the effects of these genes» mutations as possible targets for new therapeutic treatments.
In the study published in the journal Science Signaling, the team led by LLuís Espinosa, investigator of IMIM's research group into stem cells and cancer, have shown that inhibition of endosomal activity is a potential therapeutic strategy for the treatment of cancers with the BRAF mutated gene.
«Our gene therapy protocol is not yet ready for clinical trials — we need to tweak it a bit more — but in the not - too - distant future we think it could be developed for therapeutic use in humans,» says Jeffrey Holt, PhD, a scientist in the Department of Otolaryngology and F.M. Kirby Neurobiology Center at Boston Children's and an associate professor of Otolaryngology at Harvard Medical School.
Mice engineered to express Lin28 in their kidneys developed Wilms tumor, which regressed when Lin28 was withdrawn, indicating that strategies aimed at blocking or deactivating the gene hold therapeutic promise for children with Wilms.
The study paves the way for CRISPR - Cas9 as a powerful gene editing tool with potential therapeutic applications for inherited diseases — leading to more widely available gene therapy techniques.
The authors conclude that these genes may represent new therapeutic targets for HD.
Remarkably, when the researchers manipulated the activity of these three genes, almost all of the other genes in the network were corrected, pointing to novel therapeutic targets for CAVD.
A comprehensive DNA sequencing of pancreatic cancer cases revealed not only a plethora of damaged genes, but potential diagnostic biomarkers that could help identify those with longer or shorter survival, and provide opportunity for new therapeutic interventions.
«Importantly, the team was able to identify several genes that may be able to help us to predict outcomes in certain circumstances or serve as good candidates for therapeutic efforts.»
It would be very easy for a team physician to let therapeutic genes continue working for a few hours, days, or weeks after an officially sanctioned treatment ends.
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).
A new method has been found for identifying therapeutic targets in cancers lacking specific key tumor suppressor genes.
Hilbert recommends to test therapeutic phages for their ability to transfer resistance genes.
The team used gene expression profiling and found that canine B - cell lymphoma expression profiles were similar in many ways to human B - cell lymphoma, thus paving the way for future studies, including therapeutic clinical trials in dogs and humans.
It will also have to be adapted to carry genes large enough to code for many therapeutic proteins.
Their work could overcome a stumbling block for gene therapy by ensuring that a gene is actually inserted into a target cell's chromosome, where it will order the production of therapeutic proteins.
Several researchers worldwide are trying to delete disease genes for therapeutic reasons.
«Body's own gene editing system generates leukemia stem cells: Inhibiting the editing enzyme may provide a new therapeutic approach for blood cancers.»
Whilst a handful of these genes including DOT1L, BCL2 and MEN1 are already established therapeutic targets, most of them are novel and open up many new possibilities for developing effective treatments against the disease.
«A better understanding of the brain region and cell type - specific binding targets of Hnrnph1 will tell us more about the function of this gene and possibly identify new therapeutic strategies for minimizing risk and treating psychostimulant addiction — a disorder for which there is currently no FDA - approved drug,» explained corresponding author Camron Bryant, PhD, assistant professor of Pharmacology and Experimental Therapeutics & Psychiatry at BUSM.
Targeted gene therapies, however, had to wait for (1) the identification of the genes to target, (2) the cloning and / or sequencing of the relevant genes and in some cases, the specific disease - causing variant, (3) a full understanding of the normal gene function and regulation, and (4) the development of efficient ways to deliver genes to the relevant tissues at therapeutic levels.
The identification of genes that are synthetically lethal in β - catenin - activated cancer cells would provide new targets for therapeutic drug design.
This study is good example of a precision medicine themed approach to showing how a mechanistic understanding of the differential gene expression can yield biomarkers that can be used for early detection of disease, as well as potential therapeutic targets.
Since then he has continued to investigate new strategies to overcome the major hurdles to safe and effective gene transfer, translate then into new therapeutic strategies for genetic disease and cancer, and allowed new insights into hematopoietic stem cell function, induction of immunological tolerance and tumor angiogenesis.
Next we use such assays to identify LEAD chemical compound (s) with therapeutic potential for HD or to validate drug or gene - based therapeutic approaches developed by our collaborators (Pr.
The advancement of the Cas9 - based platform for screening and validation will help further the development of new therapeutic products, and Caribou's CRISPR - Cas9 technology can utilize guide RNAs specific for unique sequences and target a gene at numerous sites and therefore provide enhanced specificity.
Therapeutic applications of CRISPR - Cas gene editing are being advanced with the goals of finding cures and treatments for a range of serious diseases, developing needed medicines for both household pets and production animals, and improving cell culture systems for bioproduction.
How Parkinson's Gene may lead to New Therapeutic Strategies for Breast Cancer MyCentralJersey.com — December 1, 2017
Using tiny snippets of DNA as «barcodes,» researchers have developed a new technique for rapidly screening the ability of nanoparticles to selectively deliver therapeutic genes to specific organs of the body.
This technology helps researchers connect mutations in the so - called genomic «dark matter» with the genes they affect, potentially revealing new therapeutic targets for genetic disorders.
This call for projects «Mouse models and rare diseases» aims to give a significant boost to the development of mouse models, in order to: ◊ gain a better understanding of the pathophysiological mechanisms involved in rare diseases whose defective genes have been identified ◊ test and validate therapeutic proofs of concept, at the pre-clinical in vivo level Indeed, producing these models meets a key objective in the development of a therapeutic strategy.
The goal is to understand the role of these genes and proteins and to see if they are involved in various diseases, and thus may one day serve as viable targets for therapeutic treatments.
«In our study, we used a gene therapeutic approach, but you could purify the modified protein and inject it intravenously just like it is already done for enzyme replacement therapy,» explains Spencer.
With the reference cell census data in hand, the research team is excited to conduct additional studies, including ones involving models or human patients with gastrointestinal conditions — Crohn's disease, ulcerative colitis, gastrointestinal cancers, forms of food allergy, etc. — aimed at identifying changes in gene expression and epithelial structure and function that could reveal new insights and opportunities for therapeutic development.
PrediXcan has the potential to identify gene targets for therapeutic applications faster and with greater accuracy than traditional methods.
I look for genes, pathways, and candidate mutations associated with a disease, in order to understand the mechanism and the etiology of a disease, which may eventually lead to new therapeutic targets.
In the 1980s, Dr. Kaufman's experience with gene transfer and engineering led him to become a founding scientist at Genetics Institute Inc., where he engineered mammalian cells for high - level expression of therapeutic proteins, such as clotting factors that are now used to treat individuals with hemophilia.
Following Dr. Mendell's successful phase I trial described in the NEJM publication, additional studies to enable FDA approval of the first gene therapeutic for a fatal disease of infants are now underway.
This molecule brings a revolutionary technology platform for genetic engineering in vertebrates, including gene discovery in model species and for therapeutic transgene delivery for possible human applications.