Compositions and methods for treating neurodegenerative diseases

Abstract

Compositions and methods for treating patients with preexisting neuronal damage are disclosed. The compositions and methods use an adeno-associated virus (AAV)-based gene delivery system to deliver glial cell-derived neurotrophic factor (GDNF) to patients with neurodegenerative disorders, such as Parkinson's disease. .

Description

technical field The present invention generally relates to compositions and methods for gene delivery. In particular, the present invention relates to the delivery of glial cell line-derived neurotrophic factor (GDNF) using adeno-associated virus as a vector gene delivery system to treat pre-existing neuronal damage caused by neurodegenerative diseases, such as Parkinson's disease. Background of the invention Diseases of the central nervous system are major public health problems. Parkinson's disease (PD) affects more than one million people in the United States alone. PD is clinically manifested by decreased voluntary movement, difficulty walking, postural instability, stiffness, and tremor. PD is a progressive neurodegenerative disorder that primarily affects dopaminergic (DA) neurons in the substantia nigra (SN). Currently, a variety of central nervous system diseases are treated by systemic administration of therapeutic drugs. But systemic administration is often ine...

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Problems solved by technology

Furthermore, it is unclear whether vector-derived GDNF is retroported from axon terminals to DA neuron cell bodies in the substantia nigra

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