Innovations in oligonucleotide drug delivery.
Oligonucleotides (ONs) are a new class of therapeutic compounds under investigation for the treatment of a variety of disease states, such as cancer and HIV, and for FDA approval of an anti-CMV retinitis antisense molecule (Vitravene trade mark, Isis Pharmaceuticals). However, these molecules are limited not only by poor cellular uptake, but also by a general lack of understanding regarding the mechanism(s) of ON cellular uptake. As a result, various delivery vehicles have been developed that circumvent the proposed mechanism of uptake, endocytosis, while improving target specific delivery and/or drug stability. This review describes various traditional and novel delivery mechanisms that have been employed to improve ON cellular delivery, cost effectiveness, and therapeutic efficacy.
Lysik
Melanie A
MA
Midwestern University, College of Pharmacy-Glendale, Department of Pharmaceutical Sciences, 19555 N 59th Avenue, Glendale, Arizona 18308, USA. mlysik@Arizona.midwester.edu
Wu-Pong
Susanna
S
D000818:Animals; D016503:Drug Delivery Systems / Q000379:methods*; D006801:Humans; D009841:Oligonucleotides / Q000008:administration & dosage* / Q000737:chemistry
D009841:Oligonucleotides