Sentences with phrase «many therapeutic genes»

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.

The expression of the therapeutic gene was detected 48 hours after injection.

But the small viruses that deliver therapeutic genes can have adverse side effects at high doses.

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.

Even as researchers test these findings in animals, Nabel's team is exploring an intriguing possibility: adding the Ebola virus membrane protein to a harmless virus that could then carry therapeutic genes specifically to endothelial cells.

A carrier called a vector must be used to deliver the therapeutic gene to the patient's target cells.

The lack of discrimination in the viruses used to carry therapeutic genes has been a major obstacle to testing gene therapy in humans.

The hybrid virus is created by adding HIV RNA, a stripped - down version of the Ebola surface protein RNA and the therapeutic gene to cells.

Crucially, only the therapeutic gene is incorporated into the hybrid virus, so it can not replicate.

Finding a safe way to get therapeutic genes into the cells has been a major challenge, however.

Carrying the therapeutic genes, AdV made it into the patients» livers.

Scientists have coaxed the immune system of mice to let down its guard and allow a virus to deliver therapeutic genes.

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.

Taking advantage of this, researchers can insert therapeutic genes into a virus, then use the viruses to shuttle the genes to the appropriate cells or tissues inside a human body.

Here, the researchers delivered a therapeutic gene along with a short hairpin RNA (shRNA) into liver cells in mice.

The virus inserts the therapeutic gene into the cell's DNA and uses its instructions to produce a receptor protein — a modified version of a common glutamate receptor ion channel - that they display on their surface.

UTMD uses microscopic, gas - filled bubbles that can be paired with viral therapies, therapeutic genes and proteins, and imaging agents.

To produce an adeno - associated virus that can carry a therapeutic gene and live on its own, researchers add innocuous DNA from adenovirus during preparation.

The deck was stacked in favor of success in the choice of this particular disease since it was known that the therapeutic gene just had to be expressed in a modest number of blood cells to achieve therapeutic benefit.

This was a virus - mediated therapeutic gene to treat X-linked severe combined immunodeficiency.

Gene therapy offers the possibility of replacing the function of the defective or missing gene with that of a therapeutic gene inserted into the cell.

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.

In this trial, the therapeutic genes are transported within tiny fat bubbles, called liposomes, which can ooze through the cell membrane to deliver their genetic cargo.

«Many therapeutic genes are only expressed transiently before they get shut off by the host cell or they are not expressed as expected because they don't occupy the same position in the genome as the natural genes,» explains postdoctoral researcher and co-first author Keiichiro Suzuki.

Summary: University of Chicago researchers have developed the first practical method to limit activity of therapeutic genes to a specific cell type (smooth muscle cells), clearing a major safety hurdle facing gene therapy.

Successful application depends on two key principles: identifying an appropriate therapeutic gene and successfully delivering it to the target site while maintaining its efficacious properties (e.g., for gene expression).

Once a suitable therapeutic gene is identified, it must be effectively delivered to target cells.

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»).

B's ability to deliver potentially therapeutic genes in disease models.

Duan also provided significant expertise from a long history of work on gene therapy for neuromuscular disorders.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.

Studies are also underway evaluating therapeutic gene transfer approaches to treat cardiac and skeletal muscle dysfunction in X-linked muscular dystrophy golden retriever dogs.

Her research initially focused on the identification and characterization of juvenile dilated cardiomyopathy in Portuguese water dogs and has led to several ongoing studies, including most recently the evaluation of various candidate genes for use in a therapeutic gene transfer approach to improve cardiac function in failing hearts.

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.

For gene therapies, scientists remove most of the AAV genome, replace it with therapeutic genetic cargo, and inject trillions of copies into the patient.

A study published June 1 by Nature Communications reports scientists identify a new gene essential to this process, shedding new light on possible new therapeutic strategies.

«Neuroscientists are using these new gene - editing and molecular tools to develop potential therapeutic targets across multiple disease fronts.»

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.

Most therapeutic approaches ignore the dynamic molecular network of genes, targeting instead only very few selected disease - related genes.

«We showed that the deletion of the gene responsible for this pathway can restore the original therapeutic function of the cells,» said Prof. Leor.

Scientists have tried to harness this ability by manipulating the viral genome to remove disease - causing genes and insert therapeutic ones.

The vector then unloads its genetic material containing the therapeutic human gene into the target cell.

«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.

«Gene variants modifying Huntington's symptom onset may lead to new therapeutic strategies: Genome - wide association analysis identifies sites associated with earlier - or later - than - expected symptom appearance in human patients.»

In a one - two punch, he would deploy microorganisms to dismantle and haul out the molecular trash while delivering engineered gene and therapeutic cells to refurbish cells that have died out and gone unreplaced.

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.

Like mRNA, gene therapy can induce cells to make therapeutic proteins, but it typically introduces DNA that can integrate unpredictably into the genome.

The researchers are now investigating the effects of these genes» mutations as possible targets for new therapeutic treatments.