Sentences with phrase «gene therapy research at»

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

Not exact matches

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.

«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.»

«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.

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.

Researchers at Massachusetts Eye and Ear / Schepens Eye Research Institute have reconstructed an ancient virus that is highly effective at delivering gene therapies to the liver, muscle, and retina.

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.

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 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.»

«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.

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.

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.

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.

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

Dr George Vassiliou, joint project leader from the Sanger Institute and Consultant Haematologist at Cambridge University Hospitals NHS Trust, said: «This research has led to the identification of many potential gene targets for future AML therapy, which we are making available to other researchers to explore.

«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.»

Over at Discover magazine, Veronique Greenwood has written a terrific article explaining the development of gene therapy, one of the most promising avenues of medical research over the last several decades.

Other research at U-M is developing new options for treating brain cancer through immunotherapy — harnessing the immune system to attack cancer cells once an injection of a particular gene therapy is delivered into the brain tumor.

PNP Therapeutics, Inc. — a company based on a gene - therapy technology developed by scientists at Southern Research Institute and the University of Alabama at Birmingham (UAB)-- has found an approach that has abolished otherwise unmanageable human cancers in preclinical rodent studies.

2/12/2008 Gene Therapy Protocol at UCSD Activates Immune System in Patients with Leukemia A research team at the Rebecca and John Moores UCSD Cancer Center at University of California, San Diego (UCSD) reports that patients with chronic lymphocytic leukemia (CLL) who were treated with a gene therapy protocol began making antibodies that Gene Therapy Protocol at UCSD Activates Immune System in Patients with Leukemia A research team at the Rebecca and John Moores UCSD Cancer Center at University of California, San Diego (UCSD) reports that patients with chronic lymphocytic leukemia (CLL) who were treated with a gene therapy protocol began making antibodies thTherapy Protocol at UCSD Activates Immune System in Patients with Leukemia A research team at the Rebecca and John Moores UCSD Cancer Center at University of California, San Diego (UCSD) reports that patients with chronic lymphocytic leukemia (CLL) who were treated with a gene therapy protocol began making antibodies that gene therapy protocol began making antibodies ththerapy protocol began making antibodies that r...

Ewelina Mamcarz, M.D., presents research at the annual ASH conference that indicates that the St. Jude XSCID gene therapy has been well tolerated and effective for infants as young as 2 months old.

Oisín Biotechnology — a startup with initial funding from SENS Research Foundation, the Methuselah Foundation, and others — is close behind them, having developed a form of gene therapy that — in animals at least — also destroys these cells, using an approach that is less inherently likely to destroy healthy cells along with senescent ones.

Kevin is currently a Professor & Associate Director at the Center for Gene Therapy, City of Hope — Beckman Research Institute.

Preliminary findings indicate gene therapy pioneered at St. Jude Children's Research Hospital is safe and effective for babies with a devastating inherited disorder that leaves them with little or no immune protection

Keynote speaker Flossie Wong - Staal, one of the world's foremost authorities in the field of virology, did postgraduate work in molecular biology at UC San Diego and beginning in 1990, continued AIDS research at UCSD, working specifically in gene therapy, one of the most technologically sophisticated areas in medical research.

At RPCI, he maintains an active research program focused on finding new and efficacious therapies for cancer based on immunotherapy and gene therapy, overseeing many scientists in his research lab.

Research teams at three RPB - supported institutions have collaborated in curing color blindness with gene therapy, the second successful application of gene therapy in treating an eye disorder.

Plenary Lecture: Adaptations of the Heart: Traditional and Non-Traditional Research Approaches Leslie Leinwand, Ph.D. of the Department of Molecular, Cellular and Developmental Biology at the University of Colorado, Boulder will discuss cardiac and skeletal muscle development and function, gene therapy and cardiac genetic disease.

University of Pennsylvania immunotherapy researcher Dr. Carl June, who led the development of an experimental therapy for advanced childhood leukemia that is expected to become the first CAR T - cell therapy to win U.S. Food and Drug Administration approval, will give the keynote talk today at the Conference on Cell & Gene Therapy for HIV Cure at Fred Hutchinson Cancer Research therapy for advanced childhood leukemia that is expected to become the first CAR T - cell therapy to win U.S. Food and Drug Administration approval, will give the keynote talk today at the Conference on Cell & Gene Therapy for HIV Cure at Fred Hutchinson Cancer Research therapy to win U.S. Food and Drug Administration approval, will give the keynote talk today at the Conference on Cell & Gene Therapy for HIV Cure at Fred Hutchinson Cancer Research Therapy for HIV Cure at Fred Hutchinson Cancer Research Center.

Performs genome - wide profiling of vector integration sites as readout of cell growth at clonal level, in basic research studies and technology development, in preclinical safety studies and in gene therapy treated patients.

This latest development in gene therapy is the result of more than two decades of painstaking genetic research by Jean Bennett, MD, PhD, and Albert M. Maguire, M.D., both Professors of Ophthalmology and Co-directors of the Center for Advanced Retinal and Ocular Therapeutics at Penn Medicine.

2010 — Investigator, Institute of Laboratory Animal Sciences and Max - Planck - Partner - Group on Stem Cell Aging, Chinese Academy of Medical Sciences 2007 — 2009 Associate Investigator, Institute of Laboratory Animal Sciences and Max - Planck - Partner - Group on Stem Cell Aging, Chinese Academy of Medical Sciences 2007 Posdoc Fellow at Department of Molecular Medicine and Max - Planck - Research - Group on Stem Cell Aging, Ulm University 2005 Guest scientist at Laboratory of Stem Cell Therapy, Institute of Medical Science, University of Tokyo 2003 — 2004 Visiting Scientist at Gene Mapping Center, Max Delbr ¨ ¹ ck Center, Berlin, Germany 2001 — 2003 Research Assistant, Sino - German Laboratory for Molecular Medicine, Fu Wai Hospital & Cardiovascular Institute, Peking Union Medical College, Chinese Academy of Medical Sciences 1997 — 1998 Resident Physician, Youjian Hospital, Shangdong, China

He has authored ≥ 420 publications (h - index 75), is Editor - in Chief of The Journal of Gene Medicine, Editor of Pharmaceutical Research, board member of the German Society for Gene Therapy, committee member of the American Society of Gene and Cell Therapy (ASGCT), invited guest professor at Utrecht University (1996), Fudan University (Shanghai 2012 - 13), Sichuan University (Chengdu 2014 - 17), and was awarded with the Attocube research award, Phoenix Pharma Science award, and the election into CRS College of Research, board member of the German Society for Gene Therapy, committee member of the American Society of Gene and Cell Therapy (ASGCT), invited guest professor at Utrecht University (1996), Fudan University (Shanghai 2012 - 13), Sichuan University (Chengdu 2014 - 17), and was awarded with the Attocube research award, Phoenix Pharma Science award, and the election into CRS College of research award, Phoenix Pharma Science award, and the election into CRS College of Fellows.

In 1993, he joined the Divisions of Experimental Hematology and Bone Marrow Transplantation at St. Jude Children's Research Hospital in Memphis, where he was appointed director of the cell and gene therapy laboratories, as well as chair of the institutional review board.

Robyn Elmslie, D.V.M., veterinary oncologist at the Veterinary Referral Center (VRC) of Colorado, and her husband Steven Dow, D.V.M., Ph.D., an immunologist at the National Jewish Medical Research Center in Denver, have been evaluating gene therapy for the treatment of cancer.

Funding: This work was supported by the Alliance for Cancer Gene Therapy (R.H.V.), the Onyx and Breezy Foundation (K.U.S.), the Barry and Savannah Poodle Memorial Fund and the Mari Lowe Comparative Oncology Center (N.J.M.), the Immunobiology Program of the Abramson Cancer Center at the University of Pennsylvania (NIH grant P30 CA016520, R.H.V.), and the Oncology Research fund at the Veterinary Hospital of the University of Pennsylvania (K.U.S.).