HIGHLIGHTS
- Who: Louisa Jauze and collaborators from the Institut France have published the Article: Authorship note: LJ and MV contributed equally to this work. Conflict of interest: GR and PV are authors in patents related to the development of gene therapy approaches for GSDIII (patent no. WO/2018/162748), in the Journal: (JOURNAL)
- How: Mouse model All studies were conducted in Agl-KO mice (Agl-/-) and WT (Agl+/+) littermates.
- Future: In the next future the combination of small_molecules with improved AAV gene therapy approaches may increase safety while reducing the effective dose.
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