iPSC‐based modeling of THD recapitulates disease phenotypes and reveals neuronal malformation
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DOI: 10.15252/emmm.202215847
Abstract: Tyrosine hydroxylase deficiency (THD) is a rare genetic disorder leading to dopaminergic depletion and early-onset parkinsonism. Affected children present with either a severe form that does not respond to L-Dopa treatment (THD-B), or a milder… read more here.
Keywords: thd; ipsc dan; dan; ipsc based ... See more keywords