We report the generation of a human induced pluripotent stem cell (hiPSC) line from peripheral blood mononuclear cells of a 14-year-old male with Autism Spectrum Disorder carrying a de novo heterozygous likely-pathogenic variation c.4067C>T (p.Pro1356Leu) in Dynein Cytoplasmic 1 Heavy Chain 1 ( DYNC1H1 ) gene. Variation in this gene that encodes a motor protein may affect retrograde transport of neurotrophic signals, synaptic vesicles, organelles like golgi complex and mitochondria. Therefore, this iPSC line will be an invaluable tool to study the effects of this high-risk autism-related variant on disease manifestation and cellular functions.
Murthy et al. (2026) studied this question.