Browsing by Author "Osorio, F G"

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    • Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome 

      NOURSHARGH, S; Santiago-Fernández, O; Osorio, F G; Quesada, V; Rodríguez, F; Basso, S; Maeso, D; Rolas, L; Barkaway, A; Folgueras, A R (Nature Research, 2019-02-18)
      CRISPR/Cas9-based therapies hold considerable promise for the treatment of genetic diseases. Among these, Hutchinson–Gilford progeria syndrome, caused by a point mutation in the LMNA gene, stands out as a potential candidate. ...