02291nas a2200457   4500000000100000008004100001260001500042653001600057653001700073653004100090653003300131100001400164700002500178700002100203700001700224700001600241700002400257700001700281700001500298700001800313700001800331700001900349700002000368700001400388700001600402700001600418700001400434700001600448700001300464700002000477700001800497700001700515700001800532700002800550245012800578856007200706300001100778490000700789520102300796022001401819       2026                            d  c2026-09-0110aCRISPR-Cas910aGene Editing10ahuman induced pluripotent stem cells10aNeurodevelopmental disorders1 aCidi Chen1 aWardiya Afshar-Saber1 aIsabela Iglesias1 aKristina Kim1 aBecca Lewis1 aGayathri Srinivasan1 aCeleste Chen1 aRemi Hirsh1 aRyan Guardado1 aTaryn Polanco1 aAmanda Swanson1 aErika Norabuena1 aDosh Whye1 aAshish Jain1 aChunhui Cai1 aLiang Sun1 aMaya Chopra1 aIvy Chen1 aGrazia Iannello1 aBrian Rozumny1 aEllen Hanson1 aMustafa Sahin1 aElizabeth D. Buttermore00aDevelopment of a human iPSC and patient phenotyping resource for preclinical investigations of neurodevelopmental disorders  uhttps://www.sciencedirect.com/science/article/pii/S1873506126001698  a1040730 v953 aIn this manuscript, we report the development of a comprehensive resource designed to harness the transformative potential of patient-derived induced pluripotent stem cells (iPSCs) to advance the study of neurodevelopmental disorders (NDDs). Using CRISPR-Cas-mediated genome editing, the Human Neuron Core generated a repository comprising 29 isogenic iPSC pairs, two sex-matched parental control iPSC pairs, and one unmatched patient line representing six monogenic NDDs: Tuberous Sclerosis Complex, PTEN Hamartoma Tumor Syndrome, KCNQ2 Developmental and Epileptic Encephalopathy, FOXG1 Syndrome, Phelan-McDermid Syndrome, and SETBP1 Haploinsufficiency Disorder. In parallel, detailed clinical phenotyping data were collected to enable comparison of cellular phenotypes with clinical severity in future studies. This integrated collection of genetically defined iPSC lines and associated clinical data provides a powerful platform for investigating disease mechanisms and advancing iPSC-based drug discovery for NDDs.  a1873-5061