| Literature DB >> 33865103 |
Valeria Lucchino1, Luana Scaramuzzino2, Stefania Scalise3, Katia Grillone4, Michela Lo Conte5, Claudia Esposito6, Umberto Aguglia7, Edoardo Ferlazzo8, Nicola Perrotti9, Paola Malatesta10, Elvira Immacolata Parrotta11, Giovanni Cuda12.
Abstract
Unverricht-Lundborg disease (ULD) is an inherited form of progressive myoclonus epilepsy caused by mutations in the gene encoding Cystatin B (CSTB), an inhibitor of lysosomal proteases. The most common mutation described in ULD patients is an unstable expansion of a dodecamer sequence located in the CSTB gene promoter. This expansion is causative of the downregulation of CSTB gene expression and, consequently, of its inhibitory activity. Here we report the generation of induced pluripotent stem cell (iPSC) lines from two Italian siblings having a family history of ULD and affected by different clinical and pathological phenotypes of the disease.Entities:
Year: 2021 PMID: 33865103 DOI: 10.1016/j.scr.2021.102329
Source DB: PubMed Journal: Stem Cell Res ISSN: 1873-5061 Impact factor: 2.020