Journal
HUMAN GENE THERAPY
Volume 28, Issue 11, Pages 988-1003Publisher
MARY ANN LIEBERT, INC
DOI: 10.1089/hum.2017.160
Keywords
AAV; lentivirus and other RNA viruses; cell-based therapies; clinical trials; delivery methods
Categories
Funding
- NeurATRIS: A Translational Research Infrastructure for Biotherapies in Neurosciences
- Fondation pour la Recherche Medicale
- Bioingenierie pour la Sante [DBS20140930765]
- ParisBiotech Sante incubator
- SATT (Societe d'Acceleration de Transfert Technologique) Ile de France Innov
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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.
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