Duarte, Ana JoanaRibeiro, DiogoSantos, RenatoMoreira, LucianaBragança, JoséAmaral, Olga2020-02-142020-02-1420191873-5061http://hdl.handle.net/10400.1/13513Gaucher Disease (GD) type 3 is a neurological form of a multisystemic autosomal recessive disorder belonging to the group of lysosomal storage diseases. Causal mutations in the glucocerebrosidase 1 (GBA1) commonly lead to abnormal protein and GD, heterozygosity is a genetic risk factor for Parkinson's disease. This work describes the use of a non-integrative approach using Sendai Virus delivery to establish induced Pluripotent Stem Cells (iPSCs) from fibroblasts from a GD type 3 patient. Differentiation of iPSCs can be employed to generate a variety of complex cell types with a high degree of genetic complexity that would otherwise be unattainable.engLysosomal glucocerebrosidaseInduced pluripotent stem cells (iPSCs)Cell modelsDisease modellingSendai virusFeeder-free cultureNon-integrative vectorGaucher type 3journal articlehttps://doi.org/10.1016/j.scr.2019.101595