Cationic Polymers for the Delivery of Therapeutic Nucleotides - Nanotechnology for the Delivery of Therapeutic Nucleic Acids - page 27

Chapter 2

Cationic Polymers for the Delivery of
Therapeutic Nucleotides

Wahid Khan, Saravanan Muthupandian, and Abraham J. Domb *
School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem,
Jerusalem 91120, Israel
* [e-mail]

Gene therapy is a promising approach for treatment or prevention
of wide range of diseases associated with defective gene expression.
It involves the insertion of a therapeutic gene into cells, followed
by expression and production of the required proteins. Two main
delivery systems, including viral or non-viral gene carriers, are
currently deployed for gene therapy. Although viruses are currently
the most commonly researched vector, however, the need to resolve
drawbacks related to viral vectors (e.g., high risk of mutagenicity,
immunogenicity, low production yield, limited gene size) led to the
development of non-viral vectors

.

Several non-viral modalities are reported to transfer foreign
genetic material into cells; cationic polymers constitute one of the
most promising approach. For this reason, various cationic polymers
with diminished cytotoxicity and enhanced efficacy are rapidly
emerging as systems of choice. This chapter provides an overview
and recent developments of cationic polymers employed for in vitro