Sofia Carvalho, Luciana Moreira, Juliana Inês Santos, Paulo Gaspar, Mariana Gonçalves, Liliana Matos, Hugo David, Marisa Encarnação, Diogo Ribeiro, Ana Joana Duarte, Olga Amaral, Hugo Rocha, Luísa Diogo, Sara Ferreira, Constança Santos, Esmeralda Martins, Teresa Neuparth, Joana Soares, Marta Ribeiro, Brígida Ribeiro Pinho, Nuno Oliveira, Jorge Miguel Ascenção Oliveira, Maria João Prata, Miguel Santos, Sandra Alves, Maria Francisca Coutinho
INTRODUCTION: When it comes to disease modeling, countless models are available for Lysosomal Storage Diseases (LSD). Historically, two major approaches are well-established: in vitro assessments are performed in patient fibroblasts, while in vivo pre-clinical studies are performed in mouse models. Still, both platforms have a series of drawbacks. Thus, we implemented two alternative and innovative protocols to mimic a particular sub-group of LSDs, the Mucopolysaccharidoses both in vitro and in vivo...
November 2, 2023: Endocrine, Metabolic & Immune Disorders Drug Targets