UMass Chan Affiliations
Department of Microbiology and Physiological SystemsGene Therapy Center
Department of Ophthalmology
Document Type
Book ChapterPublication Date
2013-02-01Keywords
DependovirusGenetic Vectors
Genetic Therapy
Gene Transfer Techniques
Eye
UMCCTS funding
Genetics and Genomics
Microbiology
Molecular Genetics
Ophthalmology
Metadata
Show full item recordAbstract
Ocular gene therapy is a fast-growing area of research. The eye is an ideal organ for gene therapy since it is immune privileged and easily accessible, and direct viral delivery results primarily in local infection. Because the eye is not a vital organ, mutations in eye-specific genes tend to be more common. To date, over 40 eye-specific genes have been identified harboring mutations that lead to blindness. Gene therapy with recombinant adeno-associated virus (rAAV) holds the promise to treat patients with such mutations. However, proof-of-concept and safety evaluation for gene therapy remains to be established for most of these diseases. This unit describes the in vivo delivery of genes to the mouse eye by rAAV-mediated gene transfer and plasmid DNA electroporation. Advantages and limitations of these methods are discussed, and detailed protocols for gene delivery, required materials, and subsequent tissue processing methods are described.Source
Curr Protoc Microbiol. 2013 Feb;Chapter 14:Unit 14D.4. doi: 10.1002/9780471729259.mc14d04s28. Link to article on publisher's siteDOI
10.1002/9780471729259.mc14d04s28Permanent Link to this Item
http://hdl.handle.net/20.500.14038/29688PubMed ID
23408132Notes
First author Aditya Venkatesh is a doctoral student in the Graduate School of Biomedical Sciences (GSBS) at UMass Medical School.
Related Resources
Link to Article in PubMedae974a485f413a2113503eed53cd6c53
10.1002/9780471729259.mc14d04s28