Six induced pluripotent stem cell lines from fibroblasts of individuals with CLN3-related conditions
Summary
Primary fibroblasts from six individuals with CLN3-related conditions were used to generate induced pluripotent stem cell (iPSC) lines CHDTRi001-B, CHDTRi002-B, CHDTRi003-A, CHDTRi004-B, CHDTRi005-A, and CHDTRi006-E through the expression of four reprogramming factors: human OCT3/4, KLF4, SOX2, and c-MYC. The iPSC lines were characterized to confirm their pluripotency via immunocytochemistry, flow cytometry, and teratoma formation. Genomic stability, cell line identity, and CLN3 genotype were confirmed. These iPSC lines may be used as participant-derived experimental models for further investigation of CLN3, a rare, fatal, pediatric, blindness and neurodegenerative lysosomal disorder with no cure. Published by Elsevier B.V.
| Authors | Dwojak E, O'Mard D, Zou J, Wassif CA, Burkett S, Eckhaus M, Rueda Faucz F, Padilla C, Villasmil R, Zheng W, Dang Do AN |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2024 Dec;81:103563 |
| PubMed | 39317061 |
| PubMed Central | PMC11645225 |
| DOI | 10.1016/j.scr.2024.103563 |