Sentences with phrase «of gene therapy research»

Decades of gene therapy research has yielded a reasonably good carrier for genetic material, the adeno - associated virus (AAV).

What may sound like an ad for a futuristic tanning salon is actually a triumph of gene therapy research: A tweaked gene turns the skin cells of albino mice a dark color that lasts for weeks.

So a new wave of gene therapy research is turning to nanoparticles to get the job done.

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.

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.

Kevin Whittlesey (2006 - 07 Congressional Fellow sponsored by Optical Society of America and Materials Research Society) has moved into the gene therapy field at 4D Molecular Therapeutics as director of program and alliance management.

It's up to the scientific community to maintain and enforce international codes of ethics regarding clinical research on gene therapy techniques, the authors say.

«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.

As Saaïd Safieddine, CNRS Director of Research at the Institut Pasteur and co-senior author of the study with Prof. Christine Petit (head of the Genetics & physiology hearing unit at the Institut Pasteur), explains, «we have just shown that it is possible to partially correct a specific form of hereditary hearing loss accompanied by balance problems using local gene therapy performed after the embryogenesis of the ear, which is primarily affected by the mutation responsible for the disorder.

«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.

These annual telethons now help support four institutions: TIGEM, the Telethon Institute of Genetics and Medicine; HSR - TIGET, the San Raffaele Telethon Institute of Gene Therapy; Tecnothon, the laboratories for the creation of new equipment for the disabled; and the Dulbecco Telethon Institute, which provides research facilities for excellent young Italian scientists.

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.

«It was kind of fun being at a medical school and known as the weird guy who worked with dogs,» says Modiano, who is now a professor of comparative oncology at the University of Minnesota College of Veterinary Medicine and the Masonic Cancer Center, where his research focuses on immunology, cancer cell biology, cancer genetics, and applications of gene therapy.

«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.

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.

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.

This achievement will also contribute to accelerating the research applications of RNAi such as to the development of RNA - based next - generation drugs, for example as gene therapy to suppress the production of a disease - causing protein.

«The implications of successful gene therapy are profound, and we are delighted to be associated with this study program,» says Ernesto Bertarelli, co-chair of the Bertarelli Foundation, the primary funder of the research.

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.»

The nanotechnology approach that is being tested for inhibitor control could also improve the haemophilia treatment that is now at the cutting edge of clinical research: gene therapy.

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 research examined spinal injuries but likely has implications for treating a number of brain conditions through gene therapy targeting astrocytes, said Dr. Mark Goldberg, Chairman of Neurology & Neurotherapeutics at UT Southwestern.

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 Alliance for Cancer Gene Therapy, the Lance Armstrong Foundation, Leukemia Research Foundation, and the Scleroderma Foundation are just a few examples of the organizations supporting new investigator grants.

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.

The researchers, including scientists from The Genome Institute at Washington University School of Medicine, presented the research titled, «Patient - derived xenograft study reveals endocrine therapy resistance of ER + breast cancer caused by distinct ESR1 gene aberrations.»

New research out of the University of Michigan supports combining two approaches to fight back against gliomas: attacking the tumor with gene therapy while enhancing the immune system's ability to fight it, too.

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 Salk and TSRI scientists used a range of neuro - genetic, gene therapy, biochemical and structural biology research techniques to discover that the mutant GlyRS enzyme blocked molecular signals important for maintaining the health of motor neurons, the cells that carry messages from the brain to the muscles of the extremities.

«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.

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.

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.

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.

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: