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Gene and Cell-Based Therapies for Parkinson's Disease: Where Are We?

Accepted version
Peer-reviewed

Type

Article

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Authors

Barker, Roger A 

Abstract

Parkinson's disease (PD) is a neurodegenerative disorder that carries large health and socioeconomic burdens. Current therapies for PD are ultimately inadequate, both in terms of symptom control and in modification of disease progression. Deep brain stimulation and infusion therapies are the current mainstay for treatment of motor complications of advanced disease, but these have very significant drawbacks and offer no element of disease modification. In fact, there are currently no agents that are established to modify the course of the disease in clinical use for PD. Gene and cell therapies for PD are now being trialled in the clinic. These treatments are diverse and may have a range of niches in the management of PD. They hold great promise for improved treatment of symptoms as well as possibly slowing progression of the disease in the right patient group. Here, we review the current state of the art for these therapies and look to future strategies in this fast-moving field.

Description

Keywords

Gene therapy, adeno-associated virus, embryonic, growth factor, lentivirus, pluripotent, Cell- and Tissue-Based Therapy, Deep Brain Stimulation, Genetic Therapy, Humans, Parkinson Disease, Treatment Outcome

Journal Title

Neurotherapeutics

Conference Name

Journal ISSN

1933-7213
1878-7479

Volume Title

17

Publisher

Elsevier BV

Rights

All rights reserved
Sponsorship
Wellcome Trust (203151/Z/16/Z)
Medical Research Council (MR/R015724/1)
Medical Research Council (MC_PC_12009)
Medical Research Council (MC_PC_17230)
RAB has been supported by an NIHR Biomedical Research Award to Addenbrooke's Hospital/University of Cambridge, as well as grants from Parkinson's UK, Cure Parkinson's Trust, Rosetrees trust; CHDI, EU-FP7 programme, Michael J Fox Foundation, Evelyn Trust, NIHR i4I programme, MRC and the Wellcome trust. PCB is supported by an MRC-NIHR Clinical Academic Research Partnership Award.