Gene Therapy for Neurodegenerative Diseases
A special issue of Brain Sciences (ISSN 2076-3425).
Deadline for manuscript submissions: closed (15 January 2020) | Viewed by 15661
Special Issue Editor
Interests: neurological, neuromuscular and neurodegenerative diseases; gene therapy; AAV; in vitro modeling; lysosomal storage disorders
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Neurodegenerative disorders are a devastating burden for patients and their families. Many of these diseases progress rapidly, thereby robbing the patients of key functions including cognition, vision, hearing, and movement. There is an urgent need for the development of therapeutics to slow down or halt the progression of neurodegeneration. Gene therapy approaches using adeno-associated viral vectors (AAVs) are emerging as a promising strategy to target the nervous system and hold the potential for long-lasting benefit with only a single treatment. Many innovative strategies targeting either underlying mutations that cause neurodegeneration or interfering with pathways involved in disease progression are currently under development.
This Special Issue is dedicated to gene therapy approaches for neurodegenerative diseases. It will cover research articles on therapeutic gene therapy strategies for various disorders affecting the nervous and neuromuscular systems. Articles that describe the development of new methods or compare different viral vector systems (AAVs or other viral vectors) for nervous system use are also welcome. Comprehensive reviews providing an overview over gene therapy studies for neurodegenerative disorders or on AAV gene therapy vectors and their targeting efficiency in the nervous system will also be included.
Dr. Kathrin Meyer
Guest Editor
Manuscript Submission Information
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Keywords
- neurodegenerative disorders
- gene therapy
- adeno-associated viral vectors (AAV)
- neuromuscular disorders
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