Results 1 to 10 of about 150,411 (276)

Generation of two gene corrected human isogenic iPSC lines (NCATS-CL6104 and NCATS-CL6105) from a patient line (NCATS-CL6103) carrying a homozygous p.R401X mutation in the NGLY1 gene using CRISPR/Cas9

open access: yesStem Cell Research, 2021
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

open access: yesScience Translational Medicine, 2015
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]

open access: yesCurrent Research in Toxicology
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 [PDF]

open access: yesNature Biotechnology, 2012
exaly   +2 more sources

Neurocognitive Assessment Tools for Military Personnel With Mild Traumatic Brain Injury: Scoping Literature Review

open access: yesJMIR Mental Health, 2021
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

Mining of high throughput screening database reveals AP-1 and autophagy pathways as potential targets for COVID-19 therapeutics

open access: yesScientific Reports, 2021
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

Development and validation of PAMPA-BBB QSAR model to predict brain penetration potential of novel drug candidates

open access: yesFrontiers in Pharmacology, 2023
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

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