Search Results

81 - 90 of 247
Clinical Research
In the late 90’s and early 2000’s, scientists at the NIH highlighted an increased incidence of Parkinson disease among patients with GD and their families.
… neuronopathic, which affects the nervous system (types 2 and 3) and non-neuronopathic (type 1).  Type 1 GD: This is the most common presentation of GD in the …
Staff
Dr. Acosta is a board-certified pediatric neurologist with the NIH Undiagnosed Diseases Program's GM1 gene therapy trial.
… as the Clinical Director of the Neurofibromatosis type 1 (NF1) Program and as an associate professor at George … Baker E, Vézina G, Thurm A, Tifft CJ . GM1 Gangliosidosis Type II: Results of a 10-Year Prospective Study . medRxiv … GM1 gangliosidosis patients. Mol Genet Metab. 2023 Feb;138(2):107508. doi: 10.1016/j.ymgme.2023.107508. Epub 2023 Jan …
News Release
It’s teatime and doctors, researchers and patients are at the table. They celebrate a decade of work and launch the first in-human gene therapy trial for children with a rare and devastating disease, GM1 gangliosidosis.
News Release
Scientists drafted the blueprint for how to build a common goldfish. Now they'll use it to find out what gives ornamental goldfish their nuanced features, gaining insight into human health.
… used a tool called a PacBio RS II sequencer to read the 2 billion chemical letters in goldfish DNA. For comparison, … II sequencer allowed the researchers to rapidly read the 2 billion chemical letters in goldfish DNA. Photo credit: …
News Release
Using a virtual reality buffet, researchers studied the hundreds of small decisions mothers make while selecting food for their children.
… children's genomic risk for obesity were less subject to a type of nonconscious behavior called 'choice inertia,' or selecting the same type of food over and over again. This translated into …
News Release
NIH investigators have discovered that genomic switches of a blood cell are key to regulating the human immune system.
… if the alterations might lie in a newly discovered type of enhancer called a super-enhancer (SE). Earlier work … for the function and identity of each individual cell type. In addition, a large number of disease-associated …
News Release
TCF1 (for T cell factor-1) is essential for the creation and persistence of disease-fighting antibodies in the bloodstream.
… infection. These TFH cells then interact with another type of white blood cell - called B cells - that actually … said, the body can overproduce one or the other type of T cell, with damaging results. Failure to develop TFH …
Staff
Dr. Andy Baxevanis is a senior scientist leading NHGRI's Computational Genomics Unit.
… production being functionally linked in the same cell type of a given organism, a finding that may shed new light … attractive for study is that they possess a specific type of interstitial cell (or i-cell ) that is pluripotent, … Ctenophore Mnemiopsis leidyi and its Implications for Cell Type Evolution. Science,  342:1242592. 2013. [ PubMed ] …
News Release
NHGRI researchers have identified new genes associated with the Erdheim-Chester disease (ECD) and some possible new therapies.
… to another part of the body), ECD should be considered a type of cancer and treated by oncologists, researchers wrote. …
News Release
NHGRI researchers have identified a genomic signature for some aggressive prostate tumors, which may help healthcare providers pinpoint treatment options.