Clinical gene therapy for neurodegenerative diseases: past, present, and future

F Piguet, S Alves, N Cartier - Human gene therapy, 2017 - liebertpub.com
Human gene therapy, 2017liebertpub.com
Clinical gene therapy has made important advances over the last decade. Among
neurological diseases, severe genetic neurodegenerative conditions have been the focus of
initial clinical applications. Gene therapy has also addressed complex neurodegenerative
diseases, particularly Parkinson's disease, with encouraging results in human patients,
demonstrating that specific targeting of central nervous system (CNS) cells is a relevant
strategy for severe pathologies and that efficient access to the CNS with viral vectors is an …
Clinical gene therapy has made important advances over the last decade. Among neurological diseases, severe genetic neurodegenerative conditions have been the focus of initial clinical applications. Gene therapy has also addressed complex neurodegenerative diseases, particularly Parkinson's disease, with encouraging results in human patients, demonstrating that specific targeting of central nervous system (CNS) cells is a relevant strategy for severe pathologies and that efficient access to the CNS with viral vectors is an achievable goal. The purpose of this review is to summarize the gene therapy clinical applications that have been conducted for neurodegenerative diseases. Limitations and hurdles to obtain and demonstrate benefit in patients, and the new developments that should allow new clinical applications with high beneficial potential are discussed.
Mary Ann Liebert