Sentences with phrase «gene therapy because»

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.

Not exact matches

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.

Juan Carlos Izpisúa Belmonte of the Salk Institute in La Jolla, California, knew that stem cells are more useful for gene therapy than ordinary cells, because they produce multiple daughter cells with the modified genes.

Surprisingly, the second round of gene therapy further strengthened the brain's response to the initially treated eye, as well as the newly treated one, perhaps «because the two eyes act in concert, and some aspects of vision rely on binocularity,» she says.

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.

And because mouse embryo cells with inactivated copies of BRCA2 are more sensitive to ionizing radiation than normal cells are, «it's a reasonable extrapolation» that breast cancers with mutated copies of the gene may be especially good candidates for radiation therapy.

Nevertheless,» [the] study is very important because it demonstrates for the first time that we can use gene therapy to transform cells in the brain into ones that will secrete GDNF,» says Jeffrey Kordower, a professor of neurological sciences at Rush Presbyterian Medical Center in Chicago.

After this incident, and the death in 1999 of a young man in a clinical trial, apparently because of an immune reaction to the treatment, gene therapy research grounds nearly to a halt.

Green explains that siRNAs must be encapsulated in particles that are different from those used to carry DNA because siRNAs are about 250 times smaller than the DNA molecules usually used for gene therapy.

The United States does not have an equivalent to HFEA, but FDA has said that anyone who wants to use the technique needs to apply to the agency for permission, because MRT is a form of gene therapy.

The same process has been studied as a potential genetic therapy for more than a decade, because you can target any disease gene with matching dsRNA.

That's because stem cells must be connected to existing neural networks — something that's not yet possible — whereas gene therapy simply involves making what is left in a diseased eye photosensitive.

Tsigelny added: «The clinical treatments that do not take into consideration the specific mutations in genes, but rather treat all mutations within a specific gene such as EGFR as the same, are a «hit - or - miss» game because they are based solely on observational data on how other patients previously have reacted to drug therapies.»

Because the precise activation of Hox genes is essential for a cell's fate, «the research should prove extremely useful in developing novel embryonic stem cell - based therapies, Mazzoni adds.

A breast cancer therapy that blocks estrogen synthesis to activate cancer - killing genes sometimes loses its effectiveness because the cancer takes over epigenetic mechanisms, including permanent DNA modifications in the patient's tumor, once again allowing tumor growth, according to an international team headed by the University of Pittsburgh Cancer Institute (UPCI).

A new gene therapy treatment has restored some sight in a handful of blind patients suffering from Leber's congenital amaurosis, a syndrome in which, because of a broken or missing gene called RPE65, retinal photoreceptor cells malfunction and eventually die.

The first people to be treated with a gene therapy had ADA - SCID, also called «bubble boy disease», and some later got leukaemia, probably because the virus carrying the new genes also switched on cancer genes.

Because the vaccine causes the body to mount an immune response directed against a unique tumor, the therapy is much more effective than gene - targeted or more general chemotherapy alone.

«Because this mutation already exists in nature and is benign, this «organic gene therapy» approach should be effective and safe to use to treat, and possibly cure, serious blood disorders.

Because immunotherapies are becoming increasingly important for cancer therapy, the investigators examined genes that code for known immune targets to see if any were amplified, which may predict responsiveness to immunotherapy.

Because of the urgent needs of a large number of cancer patients, most of these gene therapy trials are directed against cancer.

«This is phenomenal, because the promise of gene therapy is clear,» he says.

Many people said they would choose not to abort an affected fetus because they had high hopes of gene therapy.

Because CRISPR will never fully be rid of off - target effects, the key question for a given therapy is not strictly how many unwanted cuts it makes, but whether it disrupts any essential genes, says Jiing - Kuan Yee, a molecular biologist at the research center City of Hope in Duarte, California.

The results drew attention in part because they come from the lab of James Wilson at the University of Pennsylvania, who led a 1999 trial in which a teenager died from an immune reaction to a different gene therapy vector.

Because RNA carries the genetic message from DNA to the cell's proteinmaking factories, or can directly perform acts such as gene regulation, it, too, is an appealing target for therapies.

Britain does not allow any form of gene therapy that alters the genetic material of future generations because of the potential for eugenics or other abuses.

But many projects come under public and federal scrutiny — the recent fracas over gene therapy trials is a case in point (Science, 24 March, p. 2163), often because the manner in which they were conducted led to concerns over the safety of the recruited participants or the protection of their confidentiality.

Gene therapy's clearest success to date has been restoring the health of about 40 children with severe combined immunodeficiency (SCID), also known as «bubble boy» disease because patients can not fight off infections and are often isolated to protect them from germs.

Doctors at Great Ormond Street say two UK patients have died in 2002, because they did not start gene therapy in time.

Because some of the genes found in the islands are known to help E.coli O157: H7 do its dirty work — by encoding for toxins and the like — they could well serve as new targets for drug therapies.

There's also a long - term risk from the gene therapy that we think is small, because we've genetically altered the T cells with a viral vector that's actually a modified, non-pathogenic form of HIV, the AIDS virus.

So that makes these killer T cells attractive for gene therapy, because they can last lifelong.

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.

«Because gene therapy may carry certain risks, we set out to see if we could perform a similar transformation with small molecules, thereby eliminating — or at least reducing — our reliance on genetic manipulation,» says Dr. Ding.

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

Gene therapy remains experimental 20 years after its first human trials because of a series of vexing problems.

Cystic fibrosis may not be the most likely candidate for gene therapy, however, because the disease affects multiple organ systems.

Because of their unique properties such as monodispersity, large surface area and high drug loading efficiency, silica nanoparticles (SiNPs) have been developed for a vast array of biomedical uses such as optical imaging, cancer therapy, targeted drug delivery and controlled drug release for genes and proteins.

There is some concern about using CRISPR - Cas9 as a therapy, because while it could delete the damaged portion from the HD gene, it could also permanently remove a part of the healthy copy.

For a quarter of a century, gene therapy has been stymied, largely because the patient's immune system attacks the treatment...

In part, that's because it's so hard to track the vehicles carrying gene therapies.

Journalist Emily Mullin: Gene therapies becoming more promising but restricted in humans because of safety concerns, prompting some researchers to...

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.

And because each cell has its own unique set of mutations, we can not solve them with the methods of gene therapy in the adult organism either today or tomorrow.

«We are really excited about this work because, short of correcting a faulty gene, protein - replacement therapy using mRNA is one of the most promising techniques we have at our disposal,» says senior author Inder Verma, professor of genetics and holder of Salk's Irwin and Joan Jacobs Chair in Exemplary Life Science.

The infomercial - level hype for both gene therapy and stem cells is not just because scientists are trying to convince funders, but because they want to believe.

«This new work is exciting, because it shows that we can directly target genes within a specific cell type, which is important for targeted therapies,» says Hedrick, a Professor in the Division of Inflammation Biology.

Rejuvenated Bio has carried out preliminary trials of gene therapy to reverse aging in beagles, claiming that if and when their process works, animals will be younger because of added DNA code and instruction to their bodies.