Sentences with phrase «gene therapy tool»

They supported a range of studies, from work on gene therapy tools (including adenovirus and rous sarcoma virus vectors) to development of a new vaccine for the deadly Marburg virus.

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.

Not exact matches

Right now, the first CRISPR gene therapies are in development, but they are not built on a «standard stack» and require a lot of custom work for each indication - these therapies will be coming into the clinic and hopefully get approved the coming couple years, and after that there could be significantly more investment into standard tools.»

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.

For his part, Collins, who has led NIH since 2009 and been kept on by the Trump administration, pointed to an array of promising NIH activities, including the development of new technologies to provide insights into human brain circuitry and function through the Brain Research through Advancing Innovative Neuroethologies (BRAIN initiative) and the use of the gene - editing tool CRISPR - Cas9 to correct mutations and clear the way to develop and test a «curative therapy» for the first molecular disease: sickle cell disease.

Study shows memories formed by the same gene - silencing tool used in embryonic development; a finding could set the stage for new therapies for schizophrenia

The study paves the way for CRISPR - Cas9 as a powerful gene editing tool with potential therapeutic applications for inherited diseases — leading to more widely available gene therapy techniques.

Many groups, including Urnov's company, are already using gene - editing tools to develop therapies that correct genetic defects in people (such as by editing white blood cells).

The new Salk tool, called CasRx, opens up the vast potential of RNA and proteins to genetic engineering, giving researchers a powerful way to develop new gene therapies as well as investigate fundamental biological functions.

It is expected that the new technique will be applied to gene therapy in the future in addition to providing a powerful tool for breeding useful organisms and conducting disease and drug - discovery research.

TMAdV's rarity in humans could make it a potentially powerful tool as a viral vehicle for delivering gene therapy, Chiu adds.

The advent of molecular cloning, DNA sequencing and the many tools of molecular genetics and cell biology has given us sufficient knowledge of the basis for disease and the genes to target, but what has limited the application of gene therapy has been efficient gene delivery systems.

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.

Adeno - associated viruses (AAVs) also remain episomal, have the ability to infect non-dividing cells, and (because of their low immunogenicity) are attractive tools for gene therapy.

, have the ability to infect non-dividing cells, and (because of their low immunogenicity) are attractive tools for gene therapy.

Embryonic stem (ES) cells are important tools in the study of gene function and may also become important in cell therapy applications.

Their findings, published last week in the Proceedings of the National Academy of Sciences, may provide clues for understanding how some forms of human lung cancer initiate and may also aid in the development of tools for successful gene therapy of lung diseases such as cystic fibrosis.

Gene - editing tool could replace antiretroviral therapy as treatment for HIV.

«The ability to direct a gene to a specific cell type and prevent expression in other cell types is a powerful new tool that allows us to bypass one of the most troubling safety concerns facing gene therapy,» said Michael Parmacek, MD, assistant professor of medicine at the University of Chicago and director of the study.

December 6, 2016 — A targeted gene - replacement technique partially restored vision in blind rats and holds promise as a potential tool for gene therapy.

Her game - changing technology takes a mysterious bacterial genetic code and transforms it into a powerful tool for cutting and pasting bits of genetic material — meaning not only could the entire field of gene therapy be revived, but her genome - editing tool could one day be used to treat a range of diseases, from cancer and AIDS to hereditary disorders like Down syndrome and Huntington disease.

Scientists seeking a simple and gentle way to provide short - term gene therapy have a new tool: nanoparticles.

Lentiviruses are rapidly gaining traction as important tools for gene and gene - modified cell therapy because of their ability to insert genes into dividing and nondividing cells.

Called excision and corrective therapy, or EXACT, this new tool acts as both a template and a bandage for repairing a malfunctioning gene.

Drawing from gene therapy, bioengineering and immunotherapy, Stephan is pinpointing the best tools in each discipline and finding new ways to combine them.

Dr. Hauk's research will define the genes associated with chronic and intermittent hypoxia to give clinicians a tool — based on the type of hypoxia — to better tailor therapy for individual patients.