Gene therapy for dopamine replacement.
Författare
Summary, in English
Dopamine replacement for Parkinson's disease (PD) have seen three major iterations of improvements since the introduction of l-3,4-dihydroxyphenylalanine (l-DOPA) pharmacotherapy: dopamine receptor agonists, ex vivo gene transfer for cell transplantation and most recently in vivo gene therapy. In this chapter, we describe the principles behind viral vector-mediated enzyme replacement in PD. We focus on the enzymes involved in the dopamine synthesis and their internal regulation, the early experimental work on gene therapy using different viral vector types and selection of transgenes, and finally discuss the recently completed early phase clinical trials in PD patients.
Avdelning/ar
Publiceringsår
2010
Språk
Engelska
Sidor
221-235
Publikation/Tidskrift/Serie
Progress in Brain Research
Volym
184
Länkar
Dokumenttyp
Artikel i tidskrift
Förlag
Elsevier
Ämne
- Neurosciences
Status
Published
Forskningsgrupp
- Molecular Neuromodulation
- Brain Repair and Imaging in Neural Systems (BRAINS)
ISBN/ISSN/Övrigt
- ISSN: 1875-7855