Corrigendum to "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" [Stem Cell Res. 56 (2021) 102554].
Functional characterization and potential therapeutic avenues for variants in the NTRK2 gene causing developmental and epileptic encephalopathies.
An induced pluripotent stem cell-derived NMJ platform for study of the NGLY1-Congenital Disorder of Deglycosylation.
An open-label study evaluating the safety, behavioral, and electrophysiological outcomes of low-dose ketamine in children with ADNP syndrome.
The precision medicine process for treating rare disease using the artificial intelligence tool mediKanren.
COVID-19 bacteremic co-infection is a major risk factor for mortality, ICU admission, and mechanical ventilation.
