Innovative & personalized RNA-based therapies for rare diseases and development of models for their testing: application to Mucolipidosis II

2026-02-112026-02-11http://hdl.handle.net/10400.18/10895, Medical and health sciences ,Medical and health sciences/Medical biotechnologyInnovative & personalized RNA-based therapies for rare diseases and development of models for their testing: application to Mucolipidosis II