17:15-18:45
Parallel 4b: Cell and gene therapy of metabolic diseases
Chair: Fatima Bosch, UAB Barcelona
17:15 Fatima Bosch, UAB Barcelona ⓘ
INV21 Gene therapy for maturity-onset diabetes of the young type 3 (MODY3)
17:45 Maria Ester Bernardo, SR Tiget, Milan
INV22 Update on the clinical trial results of HSC-gene therapy for mucopolysaccharidosis type 1 Hurler
18:15 Maria Garcia-Bravo, Division of Cell Technology, Biomedical Innovation Unit, CIEMAT, CIBERER, IIS-FJD, UAM, Madrid
OR07 Hyperoxaluric phenotype improvement after in vivo lentiviral vector gene therapy in a preclinical model of Primary Hyperoxaluria Type 1
18:30 Araceli Aguilar-González, GENyO- Centro de Genomica e Investigacion Oncologica: Pfizer / Universidad de Granada / Junta de Andalucia
OR08 Isogenic GAA-KO murine muscle cell lines mimicking severe Pompe mutations as preclinical models for screening of potential gene therapy strategies