Antisense
Chapters
Locked Nucleic Acids (LNA) and Medical Applications
Henrik Orum, Andreas Wolter and Lars Kongsbak
Much effort has been invested in altering the properties of synthetic oligonucleotides to make them more powerful tools in molecular biology, DNA based diagnostics and genomics, and as therapeutic agents. In particular, research has focused on providing DNA analogues which can enhance the affinit...
Morpholinos and PNAs Compared
James E. Summerton
This chapter will compare and contrast the properties and applications of two leading antisense molecules, Peptide Nucleic Acids (PNAs)1,2 and Morpholinos.3,4 Where appropriate, I discuss the compelling advantages which these two advanced “blocker” types provide relative to “modifier” types of...
Targeted Gene Delivery: The Role of Peptide Nucleic Acid
Kenneth W. Liang, Feng Liu and Leaf Huang
Receptor-mediated endocytosis can be exploited to achieve efficient cell-specific gene delivery. Our laboratory has used two approaches for targeted gene delivery. One uses polycation as a carrier for plasmid DNA and the other uses peptide nucleic acid (PNA) as a carrier. Targeted gene delivery usin...
The Use of PNAs and their Derivatives in Mitochondrial Gene Therapy
Paul M. Smith, Gunther F. Ross, Theresa M. Wardell, Robert W. Taylor, Douglass M. Turnbull and Robert N. Lightowlers
Human mitochondria contain their own genome, mtDNA. This small molecule encodes 24 RNA species and 13 polypeptides, which are essential components of the mitochondrial respiratory chain. The mitochondrial genome is present in hundreds or thousands of copies in each cell and is believed to turnove...
