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NGLY1 deficiency is a rare recessive genetic disease caused by mutations in the NGLY1 gene which codes for N-glycanase 1 (NGLY1). Here, we report the generation of two gene corrected iPSC lines using a patient-derived iPSC line (NCATS-CL6103) that ...
Shu Yang, Yu-Shan Cheng, Jizhong Zou
exaly +5 more sources
Financing translation: Analysis of the NCATS rare-diseases portfolio
Simulations using National Center for Advancing Translational Sciences data show that a portfolio of rare-disease therapeutics can yield attractive financial returns.
Andrew W Lo
exaly +6 more sources
Identifying androgen receptor antagonists using a metabolically competent high-throughput screening assay [PDF]
Androgen receptor (AR) is a nuclear receptor with a well-established role in sexual function and development. Modifications in AR can lead to endocrine disruption, cancer, and other diseases, making it imperative to identify compounds that influence ...
Caitlin Lynch +5 more
doaj +2 more sources
NCATS launches 'periodic table' for medicinal product ingredients [PDF]
exaly +3 more sources
BackgroundMild traumatic brain injury (mTBI) occurs at a higher frequency among military personnel than among civilians. A common symptom of mTBIs is cognitive dysfunction.
Jones, Chelsea +5 more
doaj +1 more source
Assessment - 2 What are Computerized Neurocognitive Assessment Tools (NCATs) Actually Measuring? Using Principal Component Analyses to Compare NCATs to Traditional Neuropsychological Tests [PDF]
W Cole +5 more
exaly +2 more sources
The recent global pandemic of the Coronavirus disease 2019 (COVID-19) caused by the new coronavirus SARS-CoV-2 presents an urgent need for the development of new therapeutic candidates.
Hu Zhu +9 more
doaj +1 more source
Efficiently circumventing the blood-brain barrier (BBB) poses a major hurdle in the development of drugs that target the central nervous system. Although there are several methods to determine BBB permeability of small molecules, the Parallel Artificial ...
Rintaro Kato +10 more
doaj +1 more source

