Sentences with phrase «therapeutic genes»

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.

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

Scientists have now crossed the biggest barrier to the delivery of therapeutic genes: the blood vessels.

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

By hooking up potentially therapeutic genes to this switch, the researchers hope to target treatments precisely at the melanoma and leave healthy surrounding tissue alone.

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.

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

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

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

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»).

«Combining engineered nucleases with adeno - associated viral vectors for therapeutic gene editing.»

The problem, Sweeney says, is that the viruses researchers use for delivering therapeutic genes infect primates but not other mammals.

It would be very easy for a team physician to surreptitiously let therapeutic genes continue working for a few hours, days, or weeks after an officially sanctioned treatment ends.

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.

Researchers have genetically engineered a virus that might overcome one of the big problems facing gene therapy: how to ferry therapeutic genes into target cells and keep them functioning.

The researchers want to introduce therapeutic genes into body cells that selectively kill tumour cells.

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

«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.

The prospect for successful delivery of therapeutic genes into to spinal motor neurons by intravenous injection was clear.

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 other words, therapeutic gene editing therapy must be able to efficiently edit the intended target without introducing off - target editing at sites that fortuitously resemble the intended target.

Most current gene therapy approaches rely on cold viruses, crippled so that they can't multiply within the body, to insert copies of potentially therapeutic genes into cells.

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.

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.

The generation of a functional protein product from the therapeutic gene restores the target cell to a normal state.

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

If the SM22 promoter could be linked with a therapeutic gene, such as one that curbs the proliferation of smooth muscle cells in heart arteries that have been widened in a procedure called angioplasty, the medical benefits would be enormous.

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.

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

Once the macrophages arrive in a tumor (grown in culture so far), each one releases thousands of copies of the virus, which then infect the cancer cells, after which a protein in those cells activates the therapeutic gene in each virus.

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

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

Chitosan protects the therapeutic gene product and chaperones it through the gut.

The teams led by Hélène Puccio, director of research at Inserm and Patrick Aubourg have developed a therapeutic approach based on the use of an adeno - associated virus (AAV)(1), which is known to efficiently target and express a therapeutic gene in heart cells.

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.

The authors focused on the approaches that have delivered the best outcomes in gene therapy so far: 1) direct in vivo administration of viral vectors, or the use of viruses to deliver the therapeutic genes into human cells; and 2) the transfer of genetically engineered blood or bone marrow stem cells from a patient, modified in a lab, then injected back into the same patient.

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.

New results point to therapeutic gene, not therapy itself, as cause of cancers in «bubble boy» trials

The therapeutic gene was carried by an engineered retrovirus that was used to infect the patient's blood cells before they were re-injected into the patient.

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.

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.

Dr. Verma and his colleagues have used this landmark technological development to successfully deliver the clotting factor gene to laboratory animals and to transfer a therapeutic gene to the retinal cells of mice with an inborn deficiency.