Development of polymer - lipid nanoparticles for potent mRNA delivery to the lung

Messenger RNAs (mRNAs) are an emerging therapeutic modality that holds great promise to specifically and completely treat genetic disease. mRNA has been used as a vaccine, as a protein replacement therapy, and even as a means of inducing permanent genomic editing via CRISPR. However, unlike traditional small molecule drugs, naked mRNA cannot readily enter the cellular cytosol, where it must localize in order to be successfully translated. The field of nucleic acid delivery, therefore, is largely concerned with the development of materials which can encapsulate mRNA and facilitate its transport into cellular cytoplasm in vivo. Many lipid nanoparticles originally designed to deliver short interfering RNA (siRNA) have been successfully repurposed to deliver mRNA, although their application is limited mainly to the liver. Thus, there is a continued need for the development of new materials for mRNA delivery in order to expand its therapeutic potential throughout the body.

Name thesis:degree_name

Doctoral

Department dc:contributor.department

Massachusetts Institute of Technology. Department of Chemical Engineering

Grantor dc:publisher

Massachusetts Institute of Technology

Year dc:date.issued

2018

Author dc:creator

• Kaczmarek, James Cliff.

Advisor dc:contributor.advisor

• Daniel G. Anderson.

• Chemical Engineering.

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

https://hdl.handle.net/1721.1/121895

OAI identifier oai:identifier

oai:dspace.mit.edu:1721.1/121895