scholarly journals Next-Generation Gene Therapy for Parkinson’s Disease Using Engineered Viral Vectors

2021 ◽  
pp. 1-9
Author(s):  
Tomas Björklund ◽  
Marcus Davidsson
Keyword(s):  
Parkinson’S Disease ◽  
Gene Therapy ◽  
Parkinson's Disease ◽  
Clinical Trials ◽  
Genome Editing ◽  
Viral Vectors ◽  
State Of The Art ◽  
The State ◽  
Next Generation ◽  
Suitable Vector

Recent technological and conceptual advances have resulted in a plethora of exciting novel engineered adeno associated viral (AAV) vector variants. They all have unique characteristics and abilities. This review summarizes the development and their potential in treating Parkinson’s disease (PD). Clinical trials in PD have shown over the last decade that AAV is a safe and suitable vector for gene therapy but that it also is a vehicle that can benefit significantly from improvement in specificity and potency. This review provides a concise collection of the state-of-the-art for synthetic capsids and their utility in PD. We also summarize what therapeutical strategies may become feasible with novel engineered vectors, including genome editing and neuronal rejuvenation.

Gene therapy for Parkinson’s disease using recombinant adeno-associated viral vectors

2005 ◽  
Vol 5 (5) ◽  
pp. 663-671 ◽  
Author(s):  
Shin-ichi Muramatsu ◽  
Hideo Tsukada ◽  
Imaharu Nakano ◽  
Keiya Ozawa
Keyword(s):  
Parkinson’S Disease ◽  
Gene Therapy ◽  
Parkinson's Disease ◽  
Viral Vectors

scholarly journals 200. Advancing a State of the Art Gene Therapy for Parkinson's Disease

2015 ◽  
Vol 23 ◽  
pp. S79-S80
Author(s):  
Romina Aron Badin ◽  
Katie Binley ◽  
Nadja Van Camp ◽  
Caroline Jan ◽  
Jean Gourlay ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Gene Therapy ◽  
Parkinson's Disease ◽  
State Of The Art

scholarly journals Viral vector delivery of neurotrophic factors for Parkinson's disease therapy

2015 ◽  
Vol 17 ◽  
Author(s):  
Martin J. Kelly ◽  
Gerard W. O'Keeffe ◽  
Aideen M. Sullivan
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Clinical Trials ◽  
Neurotrophic Factors ◽  
Neurotrophic Factor ◽  
Dopaminergic Neurons ◽  
Viral Vectors ◽  
Viral Vector ◽  
Neurodegenerative Disorder ◽  
The Brain

Parkinson's disease (PD) is a neurodegenerative disorder characterised by the progressive loss of midbrain dopaminergic neurons, which causes motor impairments. Current treatments involve dopamine replacement to address the disease symptoms rather than its cause. Factors that promote the survival of dopaminergic neurons have been proposed as novel therapies for PD. Several dopaminergic neurotrophic factors (NTFs) have been examined for their ability to protect and/or restore degenerating dopaminergic neurons, both in animal models and in clinical trials. These include glial cell line-derived neurotrophic factor, neurturin, cerebral dopamine neurotrophic factor and growth/differentiation factor 5. Delivery of these NTFs via injection or infusion to the brain raises several practical problems. A new delivery approach for NTFs involves the use of recombinant viral vectors to enable long-term expression of these factors in brain cells. Vectors used include those based on adenoviruses, adeno-associated viruses and lentiviruses. Here we review progress to date on the potential of each of these four NTFs as novel therapeutic strategies for PD, as well as the challenges that have arisen, from pre-clinical analysis to clinical trials. We conclude by discussing recently-developed approaches to optimise the delivery of NTF-carrying viral vectors to the brain.

Recombinant adeno-associated viral vectors bring gene therapy for Parkinson's disease closer to reality

2002 ◽  
Vol 249 (0) ◽  
pp. 1-1 ◽  
Author(s):  
Shin-ichi Muramatsu ◽  
Lijun Wang ◽  
Kunihiko Ikeguchi ◽  
Ken-ichi Fujimoto ◽  
Imaharu Nakano ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Gene Therapy ◽  
Parkinson's Disease ◽  
Viral Vectors
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