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Gene Therapy in the Management of Parkinson’s Disease: Potential of GDNF as a Promising Therapeutic Strategy

August 17, 2020

https://www.eurekaselect.com/184936/article

https://www.ncbi.nlm.nih.gov/pubmed/32811394?dopt=Abstract

TITLE:
Gene Therapy in the Management of Parkinson’s Disease: Potential of GDNF as a Promising Therapeutic Strategy.

DESCRIPTION:
Related Articles

Gene Therapy in the Management of Parkinson’s Disease: Potential of GDNF as a Promising Therapeutic Strategy.

Curr Gene Ther. 2020 Aug 17;:

Authors: Behl T, Kaur I, Kumar A, Mehta V, Zengin G, Arora S

Abstract

The limitations of conventional treatment therapies in Parkinson’s disorder, a common neurodegenerative disorder, lead to the development of an alternative gene therapy approach. Multiple treatment options targeting dopaminergic neuronal regeneration, production of enzymes linked with dopamine synthesis, subthalamic nucleus neurons, regulation of astrocytes and microglial cells and potentiating neurotrophic factors, were established. Viral vector-based dopamine delivery, prodrug approaches, fetal ventral mesencephalon tissue transplantation and dopamine synthesizing enzyme encoding gene delivery are significant therapies evidently supported by numerous trials. The review primarily elaborates the significant role of glial cell-line derived neurotrophic factor in alleviating motor symptoms and loss of dopaminergic neurons in Parkinson’s disease. Neuroprotective and neuroregenerative effects of GDNF were established via preclinical and clinical study outcomes. The binding of GDNF family ligands with associated receptors lead to the formation of a receptor-ligand complex activating Ret receptor of tyrosine kinase family, which is only expressed in dopaminergic neurons, playing an important role in Parkinson’s disease, via its association with essential protein encoded genes. Furthermore, the review establishes delivery aspects, like ventricular delivery of recombinant GDNF, intraparenchymal and intraputaminal delivery using infusion catheters. The review highlights problems and challenges of GDNF delivery, and essential measures to overcome them, like gene therapy combinations, optimization of delivery vectors, newer targeting devices, motor symptoms curbing focused ultrasound techniques, modifications in patient selection criteria and development of novel delivery strategies based on liposomes and encapsulated cells, to promote safe and effective delivery of neurotrophic factor and establishment of a routine treatment therapy for patients.

PMID: 32811394 [PubMed – as supplied by publisher]

PMID:
PubMed:32811394

DATE FOUND:
08/20/20 06:03AM

LINK / URL:
https://www.ncbi.nlm.nih.gov/pubmed/32811394?dopt=Abstract

Recent developments in the treatment of Parkinson’s Disease

July 31, 2020

https://f1000research.com/articles/9-862/v1

https://www.ncbi.nlm.nih.gov/pubmed/32789002?dopt=Abstract

TITLE:
Recent developments in the treatment of Parkinson’s Disease.

DESCRIPTION:
Recent developments in the treatment of Parkinson’s Disease.

F1000Res. 2020;9:

Authors: Stoker TB, Barker RA

Abstract

Parkinson’s disease (PD) is a common neurodegenerative disease typified by a movement disorder consisting of bradykinesia, rest tremor, rigidity, and postural instability. Treatment options for PD are limited, with most of the current approaches based on restoration of dopaminergic tone in the striatum. However, these do not alter disease course and do not treat the non-dopamine-dependent features of PD such as freezing of gait, cognitive impairment, and other non-motor features of the disorder, which often have the greatest impact on quality of life. As understanding of PD pathogenesis grows, novel therapeutic avenues are emerging. These include treatments that aim to control the symptoms of PD without the problematic side effects seen with currently available treatments and those that are aimed towards slowing pathology, reducing neuronal loss, and attenuating disease course. In this latter regard, there has been much interest in drug repurposing (the use of established drugs for a new indication), with many drugs being reported to affect PD-relevant intracellular processes. This approach offers an expedited route to the clinic, given that pharmacokinetic and safety data are potentially already available. In terms of better symptomatic therapies that are also regenerative, gene therapies and cell-based treatments are beginning to enter clinical trials, and developments in other neurosurgical strategies such as more nuanced deep brain stimulation approaches mean that the landscape of PD treatment is likely to evolve considerably over the coming years. In this review, we provide an overview of the novel therapeutic approaches that are close to, or are already in, clinical trials.

PMID: 32789002 [PubMed – as supplied by publisher]

PMID:
PubMed:32789002

DATE FOUND:
08/14/20 06:03AM

LINK / URL:
https://www.ncbi.nlm.nih.gov/pubmed/32789002?dopt=Abstract

New Molecular Drivers of Parkinson Disease Discovered, Indicating Potential Treatment Innovations

November 20, 2019

https://www.ajmc.com/newsroom/new-molecular-drivers-of-parkinson-disease-discovered-indicating-potential-treatment-innovations

New Molecular Drivers of Parkinson Disease Discovered, Indicating Potential Treatment Innovations

Phase 2 Trial of Gene Therapy, VY-AADC02, for Advanced Parkinson’s Enrolling in US

September 24, 2019

Phase 2 Trial of Gene Therapy, VY-AADC02, for Advanced Parkinson’s Enrolling in US

Phase 2 Trial of Gene Therapy, VY-AADC02, for Advanced Parkinson’s Enrolling in US

Scientists listed ways of applying genetic engineering (CRISPR) to treat Parkinson’s disease

September 10, 2019

https://www.eurekalert.org/pub_releases/2019-09/su-slw091019.php

Scientists listed ways of applying genetic engineering to treat Parkinson’s disease
How CRISPR can help fight against neurodegenerative diseases

Parkinson’s: New gene therapy shows promise for prevention

June 7, 2019

https://www.medicalnewstoday.com/articles/325407.php

Parkinson’s: New gene therapy shows promise for prevention
Published Today By Maria Cohut Fact checked by Isabel Godfrey
The hallmarks of Parkinson’s disease and some forms of dementia include Lewy bodies, toxic aggregates that form in the brain and disrupt neural circuits. Researchers from Osaka University in Japan are now testing a new preventive therapy in a preliminary mouse study.

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