Sentences with phrase «deliver therapeutic genes»

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.

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

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

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

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

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

Not exact matches

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.

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.

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.

A DNA - covered submicroscopic bead used to deliver genes or drugs directly into cells to treat disease appears to have therapeutic...

He is one of the world's leading authorities in gene therapy, having developed a gene therapy vector, based on a stripped - down version of HIV, that successfully delivers genes for therapeutic purposes.

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.

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).

Fundamental Science Overview Gene and gene - modified cell therapies treat diseases by delivering therapeutic DNA into a patient's cells, either in (in vivo) or outside his or her body (ex viGene and gene - modified cell therapies treat diseases by delivering therapeutic DNA into a patient's cells, either in (in vivo) or outside his or her body (ex vigene - modified cell therapies treat diseases by delivering therapeutic DNA into a patient's cells, either in (in vivo) or outside his or her body (ex vivo).

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

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.