Development of a U1 snRNA-adapted gene therapeutic strategy to correct 5’ splicing defects in lysosomal storage disorders

Matos, LilianaAlves, Sandra2016-03-032016-05-012015-03-19http://hdl.handle.net/10400.18/3607engDoenças GenéticasU1snRNA therapyLysosomal Storage DisordersMPS III CML III alpha/betaMPS IDevelopment of a U1 snRNA-adapted gene therapeutic strategy to correct 5’ splicing defects in lysosomal storage disordersconference object