Gene therapy for global brain diseases: one small step for mice, one giant leap for humans.

Rahim, Ahad A; Gissen, Paul · Brain · 2020

editorial · Level V

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Abstract

This scientific commentary refers to ‘Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy’, by Yoon <i>et al.</i> (doi:10.1093/brain/awaa161).

Medical subject headings