FORMULATIONS AND DELIVERY LIMITATIONS OF NUCLEIC-ACID-BASED THERAPIES

Gene delivery has expanded to include the delivery of small interfering RNA (siRNA) and oligonucleotides (ON), which can be used to decrease or downregulate the expression of a target protein. An effective gene delivery system can protect the nucleic acid from degradation, target the appropriate cell population, be efficiently internalized by the cell, avoid degradative pathways, and ultimately localize to the nucleus (DNA) or cytosol. This article focuses on systemic injection formulations, direct injection formulations, matrix-based delivery, and limitations of nonviral nucleic acid delivery.

Duke Scholars

Author Tatiana Segura Biomedical Engineering