University of Massachusetts Medical School Faculty Publications

Title

Retinal gene delivery by rAAV and DNA electroporation

UMMS Affiliation

Department of Ophthalmology; Gene Therapy Center; Department of Microbiology and Physiological Systems

Date

2-1-2013

Document Type

Book Chapter

Medical Subject Headings

Dependovirus; Genetic Vectors; Genetic Therapy; Gene Transfer Techniques; Eye

Disciplines

Genetics and Genomics | Microbiology | Molecular Genetics | Ophthalmology

Abstract

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.

Rights and Permissions

Citation: Curr Protoc Microbiol. 2013 Feb;Chapter 14:Unit 14D.4. doi: 10.1002/9780471729259.mc14d04s28. Link to article on publisher's site

Comments

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 PubMed

Keywords

UMCCTS funding

PubMed ID

23408132