Characteristics and advantages of adeno-associated virus vector-mediated gene therapy for neurodegenerative diseases.

Characteristics and advantages of adeno-associated virus vector-mediated gene therapy for neurodegenerative diseases.

Publication date: Jun 01, 2019

Common neurodegenerative diseases of the central nervous system are characterized by progressive damage to the function of neurons, even leading to the permanent loss of function. Gene therapy via gene replacement or gene correction provides the potential for transformative therapies to delay or possibly stop further progression of the neurodegenerative disease in affected patients. Adeno-associated virus has been the vector of choice in recent clinical trials of therapies for neurodegenerative diseases due to its safety and efficiency in mediating gene transfer to the central nervous system. This review aims to discuss and summarize the progress and clinical applications of adeno-associated virus in neurodegenerative disease in central nervous system. Results from some clinical trials and successful cases of central neurodegenerative diseases deserve further study and exploration.

Qu, Y., Liu, Y., Noor, A.F., Tran, J., and Li, R. Characteristics and advantages of adeno-associated virus vector-mediated gene therapy for neurodegenerative diseases. 06510. 2019 Neural Regen Res (14):6.

Concepts Keywords
Central Nervous System Gladstone Institutes
Clinical Trials Viral vector
Common Translational neuroscience
Loss Function Spinal muscular atrophy
Neurodegenerative Neurodegeneration
Neurodegenerative Diseases Senescence
Neurons Neuroscience
Progressive Neurological disorders
Regen Organ systems
Vector Branches of biology
Virus Amyotrophic lateral sclerosis
Spinal muscular atrophy
Common neurodegenerative diseases

Semantics

Type Source Name
disease DOID spinal muscular atrophy
disease MESH spinal muscular atrophy
disease MESH amyotrophic lateral sclerosis
disease DOID amyotrophic lateral sclerosis
disease DOID neurodegenerative disease
gene UNIPROT MAP6
gene UNIPROT MAGEE1
pathway BSID Neurodegenerative Diseases
disease MESH neurodegenerative diseases

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