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Defining The Potential Of Gene Therapy With Bone Morphogenetic Proteins As A Novel Therapeutic Approach In Parkinson’S Disease, Susan Rebecca Goulding Jan 2021

Defining The Potential Of Gene Therapy With Bone Morphogenetic Proteins As A Novel Therapeutic Approach In Parkinson’S Disease, Susan Rebecca Goulding

Theses

Parkinson’s disease is a neurodegenerative disorder, characterised by the progressive degeneration of midbrain dopaminergic neurons, and the intracellular aggregation of the α-synuclein protein in neurons throughout the nervous system. These changes result in the characteristic motor impairments in Parkinson’s disease. Current treatments are solely symptomatic and therefore there is an unmet clinical need to develop new disease-modifying therapies that can alter disease progression. For over 25 years, one proposed experimental therapy has focused on the delivery of proteins called neurotrophic factors to the brain to prevent dopaminergic neuron degeneration. However, to date, clinical trials using the most well-known neurotrophic factors …