Quantitative analysis of non-viral gene therapy in primary liver culture systems

Gene therapy has the potential to cure thousands of diseases caused by genetic abnormalities, provide novel combination therapies for cancers and viral infections, and offer a new and effective platform for next generation vaccines. However, after more than three decades of research and development efforts, clinical success has yet to be realized. Successful delivery of DNA is a crucial first step in attaining safe and effective gene therapeutics. While vectors based upon recombinant viruses have shown high delivery and transfection efficiencies, they may also pose certain health risks to patients, can be difficult to target to cell or tissue types of interest, and present difficulties for large-scale manufacturing. Non-viral vectors look to offer a safer alternative and can be engineered to more effectively treat a specific cell type, tissue, or pathology, but these vectors are still plagued with low transfection levels and cannot provide adequate and sustained levels of gene expression. Continued efforts focus on producing next generation non-viral vectors that safely deliver therapeutic transgenes with the efficiency of their viral counterparts. Many barriers exist in the successful trafficking of these non-viral complexes to the nucleus.

Department dc:contributor.department

Massachusetts Institute of Technology. Biological Engineering Division.

Grantor dc:publisher

Massachusetts Institute of Technology

Year dc:date.issued

2007

Author dc:creator

• Tedford, Nathan C

Advisor dc:contributor.advisor

• Linda G. Griffith and Douglas A. Lauffenburger.

• Biological Engineering Division.

Statement dc:rights

• MIT theses are protected by copyright. They may be viewed, downloaded, or printed from this source but further reproduction or distribution in any format is prohibited without written permission.

Licence dc:rights.uri

• http://dspace.mit.edu/handle/1721.1/7582

Language dc:language.iso

eng

Handle dc:identifier.uri

http://hdl.handle.net/1721.1/39914

OAI identifier oai:identifier

oai:dspace.mit.edu:1721.1/39914