Sentences with phrase «gene therapy researchers»

A problem confronting gene therapy researchers is that they can not deliver enough beneficial genes to the precise location in the body where they are needed.

The specificity of this DNA cutting activity has made CRISPR - Cas the darling of gene therapy researchers, who have modified it to make precise changes in the genomes of cultured cells, laboratory animals, and even humans.

Gene therapy researchers have found that a popular viral vector used to insert disease - curing genes into mice has a dangerous down side: it can apparently trigger liver tumors.

For nearly two decades, gene therapy researchers have focused on delivering a gene carrying the code to produce the deficient clotting factor in hemophilia.

«Several groups are working on delivering genes using this system,» says Varavani Dwarki, a gene therapy researcher at Chiron Corp. in Emeryville, California.

«It's an incredible launching pad to be able to target other diseases,» says Penn gene therapy researcher Jean Bennett, who led the study.

UPenn gene therapy researcher Jean Bennett, who heads that study (which is separate from Jacobson's) and reported the most dramatic improvements in patients» vision, says those gains seem stable for as long as 7.5 years.

The U.S. Food and Drug Administration (FDA) has begun proceedings that could disqualify gene therapy researcher James Wilson of the University of Pennsylvania in Philadelphia from conducting any future clinical trials.

According to W. French Anderson, a gene therapy researcher at the University of Southern California, severe combined immunodeficiency (SCID) was a logical target for gene therapy.

But Savio Woo, a gene therapy researcher at Mount Sinai School of Medicine in New York City and past president of the American Society of Gene Therapy, says that vigorous FDA oversight will strengthen gene therapy research.

«The technology will get better,» agrees cancer gene therapy researcher Carl June of the University of Pennsylvania.

A few years ago, gene therapy researcher Ronald Crystal and colleagues at Weill Cornell Medical College in New York City successfully slowed LINCL in mice using gene therapy in the brain.

Not exact matches

Birds, for example, can regenerate hair cells — and lately researchers have found a few similar effects in mammals that used gene and drug therapy.

James Watson, the éminence grise of gene work whose discovery of the double helix 50 years ago we are celebrating this spring, has called on his fellow researchers to show some «guts» and «try germ - line therapy without knowing if it's going to work.»

Kaplitt and his group are working with researchers at the National Institute of Mental Health in Bethesda, Md., to test the p11 gene therapy in non-human primates.

When the researchers paired female mice treated with the gene therapy with males, the females were still able to become pregnant — and have healthy babies — within the first six weeks, because of those follicles that had already started growing in the ovaries.

«Gene therapy via skin could treat many diseases, even obesity: Using CRISPR and skin grafts, researchers boost insulin levels to reduce weight.»

Sutherland says the genes she studies are already silenced in healthy adult tissue, so theoretically researchers should be able to design therapies that re-silence them in cancer cells without side effects.

While other researchers focus on using modified viruses to deliver genes for therapy, sometimes the genes are too large for viruses to carry, Lu said.

The researchers determined that CRISPR had successfully corrected a gene that causes blindness, but Kellie Schaefer, a PhD student in the lab of Vinit Mahajan, MD, PhD, associate professor of ophthalmology at Stanford University, and co-author of the study, found that the genomes of two independent gene therapy recipients had sustained more than 1,500 single - nucleotide mutations and more than 100 larger deletions and insertions.

A team of researchers at the Stanford University School of Medicine has used a gene - editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the disorder.

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 his «lessons learned» article, Wilson advises researchers against putting themselves in situations that might create potential financial conflicts (in 1992 Wilson had founded a biotechnology company focused on gene therapy).

The researchers are working with the company Oxford BioMedica to develop this idea into a gene therapy for cancer.

The new method may make some types of gene therapy easier and could be a boon for researchers hoping to control gene activity in animals, scientists say.

Using a new technique to deliver gene - therapy - like intervention directly where it's needed, researchers at Thomas Jefferson University successfully increased or decreased the muscle tone of the anal sphincter in appropriate animal models.

There's no way to cram the dystrophin gene into a virus to do traditional replacement gene therapy, but researchers have found that turning on other genes can compensate and bulk up muscles.

Since patients (and mice) with Usher 1c also have balance problems caused by hair - cell damage in the vestibular organs, the researchers also tested whether gene therapy restored balance.

Researchers at Moffitt Cancer Center hope to improve NSCLC patient survival with the results of a study that found that inherited genetic variations in interleukin genes are associated with improved patient survival and response to therapy.

The researchers assessed LPLD - related acute abdominal events that required hospital care in a small group of patients treated with a single dose of the gene therapy product Glybera ®.

Researchers are developing molecular switches that can inactivate transplanted genes, paving the way for safer gene therapies.

Researchers who previously showed that a gene therapy treatment could save the lives of dogs with a deadly disease called myotubular myopathy — a type of muscular dystrophy that affects the skeletal muscles — have found that the therapy is long - lasting.

Moreover, researchers have already developed a potential therapy that targets the gene's protein.

Researchers such as geneticist Richard King of the University of Minnesota and cell biologist Vitali Alexeev of Thomas Jefferson University are working on gene therapies or drugs that would fix albinism - causing mutations.

So the researchers decided to bypass the gene therapy approach and engineer two stable, longer - lasting forms of GDF15 that they injected directly into the animals.

With optogenetics, researchers can implant optical fibres to control genetically modified animals — could gene therapy bring it to humans?

Two of 10 children treated with gene therapy for SCID in a French trial develop leukemia, researchers announced in 2002, and it is discovered that the virus had inserted genes in several unexpected places around the genome, leading the cells to become cancerous.

Although the researchers don't know precisely how the chaperone molecule works, the difference was unmistakable: Less than 40 % of cells treated with gene therapy had clumps, compared to 70 % of controls.

The researchers found a set of frequently mutated genes in SBAs that could be helpful to clinicians when they are looking to use targeted therapies that work best in cancers with specifics mutations.

Researchers have taken a step toward changing gene therapy from a blunderbuss into a smart missile.

Researchers at the 12th International Neuroscience Winter Conference will explore, among other hot topics in neuroscience, the neurobiology of courtship, new gene therapy approaches in Parkinson's disease, the role of sleep in neuropsychiatric disorders and breakthroughs in brain repair.

In a new study, researchers are taking joint biopsy tissue from patients at the start of a new therapy and then six weeks later to see if they can find a predictor gene sequence that will clearly identify which patients respond to a particular therapy.

The researchers tested gene therapy in two types of mutant mice.

Researchers at the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children's Hospital and Houston Methodist have developed an alternative treatment in which virus - specific cells protect patients against severe, drug - resistant viral infections.

Cancer researcher Janet Sawicki of the Lankenau Institute for Medical Research in Pennsylvania, chemical engineer Robert Langer of MIT, and their colleagues are investigating biodegradable nanoparticles to deliver gene therapy for ovarian cancer.

Despite this new direction for identifying targets for pharmaceutical interventions against RP, the researchers underscore that gene therapy still has great potential and possible benefits, and they are actively pursuing efforts with this approach for several forms of RP.

This type of treatment is what we call gene therapy and researchers have been working on developing techniques to accomplish this for many years.

Her specific condition, called ADA - SCID, had long tantalized researchers seeking to repair genetic defects with a technique called gene therapy.

In an effort to expand the number of cancer gene mutations that can be specifically targeted with personalized therapies, researchers at University of California San Diego School of Medicine and Moores Cancer Center looked for combinations of mutated genes and drugs that together kill cancer cells.

With the data provided by the FLI researchers, scientists around the world may now set targeted mutations to investigate the relevance of single genes for aging, in order to better understand regulating mechanisms and, eventually, develop new approaches for therapy or prevention of aging - related diseases.