Sentences with phrase «in gene therapy»

Recent advances in gene therapies have been a boon to those researchers investigating the genetic causes of disease.

Thanks to developments in gene therapy, that day might not be so far away.

For many years, our laboratory has been one of the global leaders in the gene therapy field.

I mean, basically, there are indications that some of these things can and have caused cancer in gene therapy experiments.

One potential treatment for CF is gene therapy, and a major challenge in gene therapy is packaging replacement genes so they can be delivered to the target cells.

Even more broadly, the positive results are part of a recent trend in gene therapy success, following a shaky start more than 20 years ago.

Additionally, the scientists will investigate ways the mechanism might be applied in gene therapy.

I think, it's very promising approach in gene therapy, which eventually will enter the market.

She has been involved in executing and managing several collaborative projects in gene therapy.

When I was a postdoctoral fellow, a gene, which we had expected to potentially use in gene therapy, ended up causing horrible cancers.

And in 1999, a patient enrolled in a gene therapy trial in the United States died.

Sweeney became interested in gene therapy in 1988, shortly after scientists pinpointed the gene responsible for Duchenne muscular dystrophy.

Vectors designed and manufactured at St. Jude have already helped transform the lives of patients participating in gene therapy clinical trials for immune and blood disorders.

As a child, Zhang moved with his family from China to Des Moines, where he worked in a gene therapy lab as a high school student; the experience taught him to think about biology from an engineering point of view.

They can try to treat unwanted effects, but rescue efforts have failed in gene therapy patients [source: Raper].

Gelsinger had a rare liver disease and was participating in gene therapy research at the university when he died.

That is what killed 18 - year - old Jesse Gelsinger, who had a rare liver disease and was participating in gene therapy research at the University of Pennsylvania.

«From this knowledge, we hope to design next - generation viruses for use as vectors in gene therapy.»

«And with advances being made in gene therapy using technologies like CRISPR / Cas9, our discoveries put us closer to being able to deliver genetic «corrections» of JAGGED1 mutations.

In 2017, a steady stream of encouraging clinical results showed progress in gene therapies for hemophilia, sickle - cell disease, blindness, several serious

Because of this, families and doctors often shrink from transplant options, particularly when it comes to treating children, and it will limit the extent to which the breakthroughs in gene therapy and gene editing will be applied, explained Scadden, who is a practicing hematologist at MGH and chairman of Harvard's Department of Stem Cell and Regenerative Biology.

What's more, Discher says, unpublished work from his lab suggests that adding the molecular passports to viruses that deliver genes in gene therapy also helps them avoid immune detection.

Robillard JM, Whiteley L, Johnson TW, Lim J, Wasserman WW, Illes J. Utilizing social media to study information - seeking and ethical issues in gene therapy.

Transient liver toxicity is a significant concern in gene therapy, when high doses are required.

They then used that variant in gene therapy experiments in animals that had severe hemophilia A.

The pieces of the dead guide activators are small enough to fit in gene therapy viruses.

He says, «All of the work on structure and function and the rational design of proteins can translate into improved products offering a better chance of success in gene therapy.»

Before moving on to human trials, they will need to study all instances of «off - target» effects: Years before Crispr, the viruses employed to deliver DNA in gene therapy trials occasionally damaged the whole system, causing cancer.

Muscle biologists Qi Long Lu and Terence Partridge at the Medical Research Council Clinical Sciences Centre in London, U.K., and their colleagues decided to combined the antisense strategy with a chemical often used in gene therapy because it is known to improve delivery of DNA into cells.

It could also be relevant to clinical applications — it treats sequence uniqueness as a high priority and thus minimises the risk of potentially unwanted gene modifications, which must be avoided at all costs in gene therapy,» says Graf.

Some on the panel suggested they were particularly sensitive about such concerns given that it was at UPenn in 1999 that a young man, Jessie Gelsinger, died in a gene therapy trial, setting the field back for years.

A few years ago, a retrovirus triggered leukemia in three children in a gene therapy trial in France (ScienceNOW, 14 January 2003).

Sean Nygaard and colleagues have developed a new technique that may help to overcome one of the largest hurdles in gene therapy — the ability to generate a large pool of gene - corrected cells that would be effective in repairing or correcting injury and disease.

The light - activated genetic switch could be used to turn genes on and off in gene therapies; to turn off gene expression in future cancer therapies; and to help track and understand gene function in specific locations in the human body.

To this end, three of the paper's authors have set up AAVLife, a French company specializing in gene therapy for rare diseases, to translate to clinic these important laboratory findings.

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.

This will give way to the development of a drug to be used in gene therapy against neurodegenerative diseases based on small molecules which enhance the expression of the gene and / or the use of fragments of the Klotho protein itself.

Health authorities in several countries are responding to a serious adverse event in a gene therapy trial.

Gene therapy experts are heartened by the new results, which may counteract a wave of negative publicity after the death last year of a volunteer in a gene therapy trial at the University of Pennsylvania (ScienceNOW, 9 December 1999).

The patient was the first in a gene therapy trial to die of the therapy itself; his death is the latest blow to a field that has been struggling to live up to the promise and hype surrounding the first gene therapy trials a decade ago.

The remarkable turnaround in gene therapy is largely due to scientists» increasingly refined ability to engineer the viruses used to deliver healthy genes to the cells that need them.

Although this field is still relatively young, evidence for clinical efficacy has been observed and continued progress seems assured, as clinical trials continue to yield insights into how gene therapy can be applied and improvements are made in gene therapy tools.

The collaboration will combine Lysogene's outstanding translational and clinical expertise in gene therapy for CNS disorders with the unique preclinical expertise and infrastructure of UMMS and AU to design and test innovative AAV - based gene therapy approaches to treat GM1 - gangliosidosis.

This led the team to package the gene editing tool into AAV, used in late - phase clinical trials in the U.S. and also already approved in a gene therapy drug in the European Union.

In 1999, 18 - year - old Jesse Gelsinger became a pivotal figure in gene therapy, an unintended martyr.

Leukemia in gene therapy trials: a question of vectors, inserts and dosage?»