Gene transfer of brain derived neurotrophic factor (BDNF) prevents neurodegeneration triggered by frataxin deficiency

Y Katsu-Jiménez, F Loria, JC Corona, and J Diaz-Nido; Molecular Therapy accepted article preview 5 February 2016; doi: 10.1038/mt.2016.32

Co-injection of a herpesviral vector encoding for BDNF efficiently prevents both the development of cerebellar neuropathology and the ataxic phenotype. These data demonstrate the potential therapeutic usefulness of neurotrophins like BDNF to protect frataxin-deficient neurons from degeneration.

This work was supported by grants of the Spanish National Research Plan (SAF 2012-38042) and the Autonomous Government of Madrid (S2010/BMD-2331). Research at the authors laboratory is also supported by Friedreich Ataxia Research Alliance (FARA), Ataxia UK, FARA Ireland, Spanish FEDAES, GENEFA, Babel Family, Italian ASIA and Swedich BotaFA.

Gene transfer of brain derived neurotrophic factor (BDNF) prevents neurodegeneration triggered by frataxin deficiency