Sentences with phrase «in gene therapy research»

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

Not exact matches

Advances in molecular biology and bioinformatics have led to an explosion of research on the causes of hearing loss and how to fix it with drugs or gene therapy.

On the supply side, evolving technology and industry dynamics have driven cutting edge research and investments in diverse areas like gene therapy, early detection / diagnostics, personalized medicine and telemedicine, to better meet the demand for cost efficient and accessible healthcare.

The RAC model of diverse committee membership and open meetings has ensured a public voice in the review of the safety and ethics of gene therapy research among academic and industrial investigators.

«I think it awakens the possibility of gene therapy for neuropsychiatric diseases,» says Husseini Manji, a senior investigator at Johnson & Johnson Pharmaceutical Research & Development in Titusville, N.J., who was not involved in the study.

Although gene therapy research has made great strides in recent years, it has yet to be widely deployed, and no CRISPR - edited genes have yet been tested for safety or efficacy in human clinical trials.

«Steep funding cuts for the federal health agencies are counterproductive at a time when innovative research is moving us closer to identifying solutions for rare diseases, new prevention strategies to protect Americans from deadly and costly conditions, advances in gene therapy, new technologies for understanding the brain, and treatments that harness the ability of our immune system to fight cancer.»

The policy — in the form of first Office Actions on a series of applications for patents on expressed sequence tags, ESTs — could greatly complicate basic gene therapy research by substantially allowing patents for small sequences of a gene that may later be used by the patent holder to corner ownership and uses of entire genes.

In science news around the world, NASA's Cassini mission is about to take its final plunge into the atmosphere of Saturn after 13 years providing an unprecedented view of the planet and its moons, a fight over whether to preserve or develop of one Europe's oldest gold mining sites heats up again, the U.S. Food and Drug Administration approves the first cancer gene therapy for people, a U.S. court gives a green light to a $ 1 billion lawsuit brought by the Guatemalan victims and survivors of mid — 20th century syphilis experiments by research institutions including Johns Hopkins University, and more.

There already has been great effort to elucidate the mechanisms of gene expression regulation, but it will be important to continue research to fully realize the potential of these epigenetic therapies and their applications in clinical settings.

«Genes may cause tumor aggressiveness, drug resistance in African - American prostate cancer: Research found many targeted therapies for prostate cancer may not be effective against tumors in African - American men.»

These include the ability to bring new, innovative products to the market; progress in oncology, such as the approval of Genentech's drug Avastin for breast cancer and advances in the use of gene therapy, despite some setbacks; continuing progress in research on stem cells; the emergence of treatments for previously untreated diseases; and solutions for food and fuel shortages, such as biocrops and biofuels.

«The bottom line question,» says Christopher Austin, a neurogeneticist at Merck Research Laboratories in West Point, Pennsylvania, is whether this kind of gene therapy can stem the rate of brain cell death.

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.

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.

In the midst of these investigations, gene therapy research almost ground to a complete halt.

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.

Since we have succeeded in confirming the accuracy of CRISPR - Cas9, we anticipate that there will be a great progress in the development of gene or cell therapies,» emphasizing the significance of this research achievement.

«Research into basic workings of immune system points to way of improving therapies for cancer: Differences in wiring of «exhausted» and effective T cells indicate possible gene - editing targets.»

Independent studies by Hortelano's group and other research teams in Germany and the United States have shown that this oral gene therapy does not activate the immune system.

Although the gene / cell therapy strategy was highly successful in laboratory mice, the authors stressed that additional research and testing are needed before the therapy could be tested in humans.

To prevent this decline in Area X, White's research team used methods similar to human gene therapy to insert a version of FoxP2 in male zebra finches.

The finding warrants research into adding drugs that could prevent the cancer from hijacking patients» repressive gene regulatory machinery, which might allow the original therapy to work long enough to eradicate the tumor, the researchers report in their National Institutes of Health - funded study, published in the current issue of Science Translational Medicine.

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.

The roadmap outlines future research directions toward the goal of enhancing human radioresistance, including upregulation of endogenous repair and radioprotective mechanisms, possible leeways into gene therapy in order to enhance radioresistance via the translation of exogenous and engineered DNA repair and radioprotective mechanisms, the substitution of organic molecules with fortified isoforms, the coordination of regenerative and ablative technologies, and methods of slowing metabolic activity while preserving cognitive function.

We hope that further research may identify why a faulty INPP5K gene causes these symptoms — and that information will help with the search for therapies in the future.»

In a long - awaited success for gene therapy, a research team from the University of Pennsylvania restored sight to two patients suffering from Leber's Congenital Amaurosis (LCA), a previously untreatable form of blindness.

«The study showed for the first time that a modified nNOS gene could be delivered through gene therapy to protect the hearts of mice from Duchenne muscular dystrophy,» said Dongsheng Duan, PhD, co-author of the study and Margaret Proctor Mulligan Professor in Medical Research at the MU School of Medicine.

The team at UF's Powell Center for Rare Disease Research and Therapy conducted the first in - human study of gene therapy to treat respiratory dysfunction in patients with infantile onsetTherapy conducted the first in - human study of gene therapy to treat respiratory dysfunction in patients with infantile onsettherapy to treat respiratory dysfunction in patients with infantile onset Pompe.

In the same week that the trial was approved, the Medical Research Council announced that it was to set up its own committee to oversee its work in gene therapIn the same week that the trial was approved, the Medical Research Council announced that it was to set up its own committee to oversee its work in gene therapin 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.

Formerly a research leader at CHOP, High pursued groundbreaking preclinical investigations in hemophilia B gene therapy and provided scientific expertise to previous gene therapy trials in hemophilia and other genetic disorders at CHOP before moving to Spark Therapeutics, which was spun off from CHOP in 2013.

They do social science, health, philosophical, policy, or legal research on topics such as privacy, confidentiality, the psychological impact of genetic information, informed - consent issues in genomics research, commercialization of genetic products, genetically modified foods, behavioral genetics, gene testing, and gene therapy.

Many in medical research now fear that the infamous and discredited «no patents, no cure» lobbying slogan for patented genes may now mean therapies too expensive for the patients it was claimed to cure.

Researchers from the Institute of Neurosciences at the Universitat Autònoma de Barcelona (INc - UAB) and the Vall d'Hebron Research Institute (VHIR) are the first to demonstrate that regulation of the brain's Klotho gene using gene therapy protects against age - related learning and memory problems in mice.

The research team found that when a particular mitochondrial fission protein (GTPase dynamin - related protein - 1 — Drp1) was blocked using either gene - therapy or a chemical approach in experimental models of PD in mice, it reduced both cell death and the deficits in dopamine release — effectively reversing the PD process.

Secondly, this is the first research to demonstrate that the loss of hair cells and hearing can be curtailed in USH3 mice by clarin - 1 gene therapy.

Dr. Giatsidis and coauthors reviewed the state of the art in research on gene therapy techniques for treatment of local disorders and injuries — the first such review in more than a decade.

Several research groups are pursuing gene therapy approaches to regenerate skin, such as using genes to control expression of growth factors involved in skin healing.

This conference touched on a broad spectrum of topics encompassing scientific integrity, including gene therapy, guidelines for animal and human subject use, authorship, public health issues, and the involvement of minorities in research.

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 reseagene 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 retherapy 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 reseaGene Therapy, says that vigorous FDA oversight will strengthen gene therapy reTherapy, says that vigorous FDA oversight will strengthen gene therapy reseagene therapy retherapy research.

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.

Next Wave asked: If research on gene vectors can be used not only to further gene therapy but also — potentially — to aid in the development of biological weapons, should scientists be concerned about the potential applications of their research in genetics (as Joseph Rotblat suggested in a 19 Nov 1999 editorial in Science magazine?)

Arthur Nienhuis, a hematologist at St. Jude Children's Research Hospital in Memphis, Tenn., and president of the American Society of Gene Therapy, responds:

The research, part of a phase I clinical trial to test the safety of the treatment, was published as a letter to the editor in The New England Journal of Medicine earlier this week and will be in the September issue of Human Gene Therapy.

The research suggests the possibility, Kahn explains, of developing gene therapy treatments using fat cells that aid in treating metabolic diseases, cancer or other conditions in the liver or other organs.

«More research is being conducted with VNA gene therapy and it's hard to deny the potential of this rapid - acting and long - lasting therapy in treating these and several other important illnesses.»

«RNAi therapies are a unique approach to cancer treatment as they have the potential to «turn off» the genes» coding for proteins involved in cancer cell division,» said Ramesh K. Ramanathan, M.D., medical director of the Virginia G. Piper Cancer Center Clinical Trials Program at Scottsdale Healthcare and deputy director of the Clinical Translational Research Division of the Translational Genomics Research Institute (TGen) in Phoenix, Ariz. «Using a lipid nanoparticle, the RNAi drug can be delivered to a cancer cell to block the expression of specific proteins involved in tumor growth.»

Their research has created the technology known as gene targeting, now used in virtually all areas of biomedicine — from basic research to the development of new therapies.

BETHESDA, Md., Mon., Jan. 26, 2004 - A Japanese - U.S. team today reported the successful creation of transgenic animals using sperm genetically modified and grown in a laboratory dish, an achievement with implications for a wide range of research from developmental biology to gene therapy.