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Monday 30 May

Room: Ibiza

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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


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