Viral Vectors Paired with Genome-Editing Tools: Evolving Technologies for the Treatment of Neurodegenerative Diseases and Disorders
A special issue of Cells (ISSN 2073-4409). This special issue belongs to the section "Cell Methods".
Deadline for manuscript submissions: closed (31 May 2022) | Viewed by 232
Special Issue Editors
Interests: gene therapy; neurodegenerative diseases; gene regulation; epigenetic
Special Issues, Collections and Topics in MDPI journals
Interests: age-related neurodegenerative diseases; neurogenetics; functional genomics; regulation of gene expression; precision medicine and ‘next generation’ gene therapy approaches for Alzheimer’s-Parkinson’s
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Diseases and disorders of the central nervous system (CNS) have historically been among the most difficult to combat using conventional pharmacological strategies. Emerging technologies based on ZFs, TALEs, and CRISPR/Cas systems have greatly extended the boundaries of genome manipulation within the CNS and promoted genome editing approaches to the level of promising strategies for counteracting many brain diseases. The parallel development of efficient viral and non-viral delivery systems has also significantly increased our access to the treatment options for CNS diseases. In this chapter, we describe the various tools available for genome editing and summarize in vitro and in vivo preclinical studies of CNS genome editing. We discuss the future progress of these technologies, as well as the current limitations and alternative approaches to overcoming some of their bottlenecks. A special emphasis will be put into viral-mediated gene transfer represents an attractive mean of the delivery of therapeutic cargo into the CNS. As such, we will address in detail the current progress put towards the development of improved vector systems for gene- and epigenome-editing tools into the CNS. Specifically, we will aim here to devote significant attention to viral vectors derived from human immunodeficiency virus type 1 (lentiviral vectors; LVs) and adeno-associated virus (AAVs). The high interest in these viral delivery systems vectors is due to: (i) robust delivery and long-lasting expression; (ii) efficient transduction into postmitotic cells, including the brain; (iii) low immunogenicity and toxicity; and (iv) compatibility with advanced manufacturing techniques. Finally, this chapter will summarize the current therapeutic application and novel approaches of advance genome-editing technology for the prevention and treatment of neurodegenerative diseases (NDDs).
Dr. Boris Kantor
Prof. Dr. Ornit Chiba-Falek
Guest Editors
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Keywords
- gene editing
- epigenome editing
- CRISPR/Cas
- CNS diseases
- viral vectors
