Sentences with phrase «for gene therapy research»

That clearance would be a high - water mark for gene therapy research, which suffered a major setback in 1999 with the death of a patient in a clinical trial for a liver disorder.

The climate for gene therapy research has since begun a slow rebound.

Not exact matches

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.

She has served as a biochemical patent agent and a research scientist for a gene - therapy company.

Out Run Rett raises funds for promising research involving gene therapy and honors two young Southold residents, Maren and Rachel, who suffer from the disorder.

«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.

«While much work remains to be done to develop this research into clinical applications, our findings could have a promising translational impact, both for protein replacement and gene therapy,» said Arruda.

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.

The Food and Drug Administration immediately terminated all gene therapy trials there, and the incident prompted federal regulators to establish new rules for human gene therapy research.

«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.

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.

«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.

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 diseaFor 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 diseafor the first molecular disease: sickle cell disease.

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.

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.

«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.

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.

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.

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 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.

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.

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.

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.

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.

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.

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.»

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.

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.

As gene therapy and CRISPR / Cas9 - style gene editing research progresses, we can anticipate demand for services such as those provided by the Viral Vector Core.

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AD 129 — c. 216) galena GALEX (Galaxy Evolution Explorer) Galilean satellites Galilean telescope Galileo (Galilei, Galileo)(1564 — 1642) Galileo (spacecraft) Galileo Europa Mission (GEM) Galileo satellite navigation system gall gall bladder Galle, Johann Gottfried (1812 — 1910) gallic acid gallium gallon gallstone Galois, Évariste (1811 — 1832) Galois theory Galton, Francis (1822 — 1911) Galvani, Luigi (1737 — 1798) galvanizing galvanometer game game theory GAMES AND PUZZLES gamete gametophyte Gamma (Soviet orbiting telescope) Gamma Cassiopeiae Gamma Cassiopeiae star gamma function gamma globulin gamma rays Gamma Velorum gamma - ray burst gamma - ray satellites Gamow, George (1904 — 1968) ganglion gangrene Ganswindt, Hermann (1856 — 1934) Ganymede «garbage theory», of the origin of life Gardner, Martin (1914 — 2010) Garneau, Marc (1949 ---RRB- garnet Garnet Star (Mu Cephei) Garnet Star Nebula (IC 1396) garnierite Garriott, Owen K. (1930 ---RRB- Garuda gas gas chromatography gas constant gas giant gas laws gas - bounded nebula gaseous nebula gaseous propellant gaseous - propellant rocket engine gasoline Gaspra (minor planet 951) Gassendi, Pierre (1592 — 1655) gastric juice gastrin gastrocnemius gastroenteritis gastrointestinal tract gastropod gastrulation Gatewood, George D. (1940 ---RRB- Gauer - Henry reflex gauge boson gauge theory gauss (unit) Gauss, Carl Friedrich (1777 — 1855) Gaussian distribution Gay - Lussac, Joseph Louis (1778 — 1850) GCOM (Global Change Observing Mission) Geber (c. 720 — 815) gegenschein Geiger, Hans Wilhelm (1882 — 1945) Geiger - Müller counter Giessler tube gel gelatin Gelfond's theorem Gell - Mann, Murray (1929 ---RRB- GEM «gemination,» of martian canals Geminga Gemini (constellation) Gemini Observatory Gemini Project Gemini - Titan II gemstone gene gene expression gene mapping gene pool gene therapy gene transfer General Catalogue of Variable Stars (GCVS) general precession general theory of relativity generation ship generator Genesis (inflatable orbiting module) Genesis (sample return probe) genetic code genetic counseling genetic disorder genetic drift genetic engineering genetic marker genetic material genetic pool genetic recombination genetics GENETICS AND HEREDITY Geneva Extrasolar Planet Search Program genome genome, interstellar transmission of genotype gentian violet genus geoboard geode geodesic geodesy geodesy satellites geodetic precession Geographos (minor planet 1620) geography GEOGRAPHY Geo - IK geologic time geology GEOLOGY AND PLANETARY SCIENCE geomagnetic field geomagnetic storm geometric mean geometric sequence geometry GEOMETRY geometry puzzles geophysics GEOS (Geodetic Earth Orbiting Satellite) Geosat geostationary orbit geosynchronous orbit geosynchronous / geostationary transfer orbit (GTO) geosyncline Geotail (satellite) geotropism germ germ cells Germain, Sophie (1776 — 1831) German Rocket Society germanium germination Gesner, Konrad von (1516 — 1565) gestation Get Off the Earth puzzle Gettier problem geyser g - force GFO (Geosat Follow - On) GFZ - 1 (GeoForschungsZentrum) ghost crater Ghost Head Nebula (NGC 2080) ghost image Ghost of Jupiter (NGC 3242) Giacconi, Riccardo (1931 ---RRB- Giacobini - Zinner, Comet (Comet 21P /) Giaever, Ivar (1929 ---RRB- giant branch Giant Magellan Telescope giant molecular cloud giant planet giant star Giant's Causeway Giauque, William Francis (1895 — 1982) gibberellins Gibbs, Josiah Willard (1839 — 1903) Gibbs free energy Gibson, Edward G. (1936 ---RRB- Gilbert, William (1544 — 1603) gilbert (unit) Gilbreath's conjecture gilding gill gill (unit) Gilruth, Robert R. (1913 — 2000) gilsonite gimbal Ginga ginkgo Giotto (ESA Halley probe) GIRD (Gruppa Isutcheniya Reaktivnovo Dvisheniya) girder glacial drift glacial groove glacier gland Glaser, Donald Arthur (1926 — 2013) Glashow, Sheldon (1932 ---RRB- glass GLAST (Gamma - ray Large Area Space Telescope) Glauber, Johann Rudolf (1607 — 1670) glaucoma glauconite Glenn, John Herschel, Jr. (1921 ---RRB- Glenn Research Center Glennan, T (homas) Keith (1905 — 1995) glenoid cavity glia glial cell glider Gliese 229B Gliese 581 Gliese 67 (HD 10307, HIP 7918) Gliese 710 (HD 168442, HIP 89825) Gliese 86 Gliese 876 Gliese Catalogue glioma glissette glitch Global Astrometric Interferometer for Astrophysics (GAIA) Global Oscillation Network Group (GONG) Globalstar globe Globigerina globular cluster globular proteins globule globulin globus pallidus GLOMR (Global Low Orbiting Message Relay) GLONASS (Global Navigation Satellite System) glossopharyngeal nerve Gloster E. 28/39 glottis glow - worm glucagon glucocorticoid glucose glucoside gluon Glushko, Valentin Petrovitch (1908 — 1989) glutamic acid glutamine gluten gluteus maximus glycerol glycine glycogen glycol glycolysis glycoprotein glycosidic bond glycosuria glyoxysome GMS (Geosynchronous Meteorological Satellite) GMT (Greenwich Mean Time) Gnathostomata gneiss Go Go, No - go goblet cell GOCE (Gravity field and steady - state Ocean Circulation Explorer) God Goddard, Robert Hutchings (1882 — 1945) Goddard Institute for Space Studies Goddard Space Flight Center Gödel, Kurt (1906 — 1978) Gödel universe Godwin, Francis (1562 — 1633) GOES (Geostationary Operational Environmental Satellite) goethite goiter gold Gold, Thomas (1920 — 2004) Goldbach conjecture golden ratio (phi) Goldin, Daniel Saul (1940 ---RRB- gold - leaf electroscope Goldstone Tracking Facility Golgi, Camillo (1844 — 1926) Golgi apparatus Golomb, Solomon W. (1932 — 2016) golygon GOMS (Geostationary Operational Meteorological Satellite) gonad gonadotrophin - releasing hormone gonadotrophins Gondwanaland Gonets goniatite goniometer gonorrhea Goodricke, John (1764 — 1786) googol Gordian Knot Gordon, Richard Francis, Jr. (1929 — 2017) Gore, John Ellard (1845 — 1910) gorge gorilla Gorizont Gott loop Goudsmit, Samuel Abraham (1902 — 1978) Gould, Benjamin Apthorp (1824 — 1896) Gould, Stephen Jay (1941 — 2002) Gould Belt gout governor GPS (Global Positioning System) Graaf, Regnier de (1641 — 1673) Graafian follicle GRAB graben GRACE (Gravity Recovery and Climate Experiment) graceful graph gradient Graham, Ronald (1935 ---RRB- Graham, Thomas (1805 — 1869) Graham's law of diffusion Graham's number GRAIL (Gravity Recovery and Interior Laboratory) grain (cereal) grain (unit) gram gram - atom Gramme, Zénobe Théophile (1826 — 1901) gramophone Gram's stain Gran Telescopio Canarias (GTC) Granat Grand Tour grand unified theory (GUT) Grandfather Paradox Granit, Ragnar Arthur (1900 — 1991) granite granulation granule granulocyte graph graph theory graphene graphite GRAPHS AND GRAPH THEORY graptolite grass grassland gravel graveyard orbit gravimeter gravimetric analysis Gravitational Biology Facility gravitational collapse gravitational constant (G) gravitational instability gravitational lens gravitational life gravitational lock gravitational microlensing GRAVITATIONAL PHYSICS gravitational slingshot effect gravitational waves graviton gravity gravity gradient gravity gradient stabilization Gravity Probe A Gravity Probe B gravity - assist gray (Gy) gray goo gray matter grazing - incidence telescope Great Annihilator Great Attractor great circle Great Comets Great Hercules Cluster (M13, NGC 6205) Great Monad Great Observatories Great Red Spot Great Rift (in Milky Way) Great Rift Valley Great Square of Pegasus Great Wall greater omentum greatest elongation Green, George (1793 — 1841) Green, Nathaniel E. Green, Thomas Hill (1836 — 1882) green algae Green Bank Green Bank conference (1961) Green Bank Telescope green flash greenhouse effect greenhouse gases Green's theorem Greg, Percy (1836 — 1889) Gregorian calendar Grelling's paradox Griffith, George (1857 — 1906) Griffith Observatory Grignard, François Auguste Victor (1871 — 1935) Grignard reagent grike Grimaldi, Francesco Maria (1618 — 1663) Grissom, Virgil (1926 — 1967) grit gritstone Groom Lake Groombridge 34 Groombridge Catalogue gross ground, electrical ground state ground - track group group theory GROUPS AND GROUP THEORY growing season growth growth hormone growth hormone - releasing hormone growth plate Grudge, Project Gruithuisen, Franz von Paula (1774 — 1852) Grus (constellation) Grus Quartet (NGC 7552, NGC 7582, NGC 7590, and NGC 7599) GSLV (Geosynchronous Satellite Launch Vehicle) g - suit G - type asteroid Guericke, Otto von (1602 — 1686) guanine Guiana Space Centre guidance, inertial Guide Star Catalog (GSC) guided missile guided missiles, postwar development Guillaume, Charles Édouard (1861 — 1938) Gulf Stream (ocean current) Gulfstream (jet plane) Gullstrand, Allvar (1862 — 1930) gum Gum Nebula gun metal gunpowder Gurwin Gusev Crater gut Gutenberg, Johann (c. 1400 — 1468) Guy, Richard Kenneth (1916 ---RRB- guyot Guzman Prize gymnosperm gynecology gynoecium gypsum gyrocompass gyrofrequency gyropilot gyroscope gyrostabilizer Gyulbudagian's Nebula (HH215)

This also holds true for the development of new therapeutics (vaccines, gene therapy) and transplantation research.

The donation will support an innovative program with three leading medical research groups and, for the first time, will introduce gene editing and stem cell therapies to effect treatments and, eventually, a potential cure for Huntington's disease.

Gene Therapy Approaches Studied for «Almost Every Tissue»... 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 decGene Therapy Approaches Studied for «Almost Every Tissue»... 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 Therapy Approaches Studied for «Almost Every Tissue»... 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 decgene therapy techniques for treatment of local disorders and injuries — the first such review in more than a therapy techniques for treatment of local disorders and injuries — the first such review in more than a decade.

Lysogene is a pioneer in the basic research and clinical development of gene therapy for neurodegenerative disorders.

Luigi Naldini), Gene and neural stem cell therapy for LSD Unit 2001 - 2005 Research Scientist, Stem Cell Research Institute (SCRI; Dir.

You can send it to us via our Facebook page or through the contact form... Continue reading Research Roundup: gene therapy for inherited blindness, urea implicated as a major cause of dementia and more!

To support work that can significantly contribute to a better understanding of the gene / protein leucine - rich repeat kinase 2 (LRRK2), its relevance to Parkinson's disease (PD) and effective ways to translate LRRK2 research into therapies for patients.

The present research provides a theoretical backbone for gene introduction research previously conducted by trial - and - error, and is expected to contribute to the elucidation of the molecular basis of DNA medicines and gene therapy, and to make them more efficient.

Still, BioViva has pressed ahead with telomerase gene therapy, and factions within the research community are also aiming for the same outcome of human tests, though through more conventional channels.