Parkinson's Disease PD
Adeno-associated viral vectors (AAVs) have been demonstrated to be safe and effective gene delivery system in preclinical and clinical gene therapies of Parkinson's disease (PD). As a leading service provider in the field of gene therapy, Creative Biolabs has a professional expert team with extensive experience and advanced technology platforms to offer a full range of AAV design and engineering services for the gene therapy of PD.
Introduction of Parkinson's Disease Treatment
As a common neurological disease, PD is a long-term disorder that mainly occurs in elder individuals over the age of 60. PD is a kind of progressive neurodegenerative disease that affects the motor system leading to varying degrees of dyskinesia (e.g., static tremor, motor retardation, muscle stiffness) and mental illness (e.g., depression, anxiety, dementia, cognitive impairment). The exact pathogenesis of PD is not yet so clear, which may be caused by the degeneration/death of dopaminergic neurons in substantia nigra resulting from inheritance, aging, environmental factors, oxidative stress, etc.
Despite no cure for PD, enormous devotion and progress have been made in the treatment of PD. Medication is an efficient therapy for controlling PD symptoms, of which the commonly used drugs include levodopa, dopamine agonists, monoamine oxidase inhibitors, and Catechol-O-methyl-transferase inhibitors. All these medications are less effective as with the disease progresses, and even cause side effects and complications. Recently, novel effective therapies, especially gene therapy, have been developed for the PD treatment, which may bring good news to PD patients.
AAV Vector-Based Gene Therapy for Parkinson's Disease
Gene therapy for PD treatment is an approach with great promise and advantages, whose vector delivery system can effectively penetrate the blood-brain barrier (BBB) reaching the brain lesions to play therapeutic roles. AAVs are the most frequently used viral vectors for the PD gene therapy in clinical trials due to their non-pathogenicity, low-toxicity, and good neuronal tropism. Therapeutic genes are transferred by AAV vectors into neurons for the PD treatment via compensating the loss of dopamine (a neurotransmitter) or protecting the dopaminergic neurons from degeneration/death.
One method of AAV vector-based gene therapies for the PD treatment is to control symptoms by compensating for the dysfunction caused by the degeneration of the dopaminergic neurons, for instance, the delivery of genes (TH, AADC, and GCH1) to induce ectopic dopamine synthesis. Another approach is disease modification focusing on the delivery of neurotrophic factors (GDNF) gene, which is an astrocyte-secreted protein protecting the dopaminergic neuronal functions.
Figure 1. AAV vector-based gene therapy for Parkinson's disease. (Vats, 2016)
As a leading services provider in gene therapy, Creative Biolabs is proud to provide different AAV vector products and design services for PD treatment. Our proven and optimized GTOnco™ platforms can help you quickly get satisfactory results without repeated trials. We also provide custom services based on the requirements of the clients to meet the specific demand. Please contact us for more information.
Reference
- Vats, S.; Rathod, B. (2016). Gene Therapy for Parkinson's Disease. International Journal of Scientific & Engineering Research. 9(7): 273-283.
