Abstract
The effective delivery of gene therapy can be reduced to three essential components: (1) specific targeting of an anatomical region or cell population, (2) transfer of genetic material to the host, and (3) efficacious expression that alters the disease phenotype. The predominant delivery methods are viral-mediated and nonviral-mediated therapies. Each method has benefits and caveats that affect the targeting of specific regions, stable or unstable transfer of genetic material, and effect on the targeted disorder. In this chapter we will discuss viral- and nonviral-mediated therapies, challenges in applying these therapies in animal models and human patients, and ways to circumvent these delivery roadblocks. We will also discuss specific adaptations of these methods in attempting delivery to the central nervous system.
Original language | English (US) |
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Title of host publication | Nervous System Drug Delivery |
Subtitle of host publication | Principles and Practice |
Publisher | Elsevier |
Pages | 447-461 |
Number of pages | 15 |
ISBN (Electronic) | 9780128139981 |
ISBN (Print) | 9780128139974 |
DOIs | |
State | Published - Jun 25 2019 |
Keywords
- CRISPR/Cas9
- Central nervous system
- Gene therapy
- Stem cells
- Stereotactic surgery
- Virus
ASJC Scopus subject areas
- General Medicine