Transfection and Intracellular Drug Delivery Sytems
Many pharmaceutical agents, including various large molecules such as nucleic acids, proteins, enzymes, antibodies or drug-loaded pharmaceutical nanocarriers, need to be delivered intracellularly to exert their therapeutic action.
This intracellular transport of different biologically active molecules is one of the key problems in drug delivery. Biological membranes prevent these hydrophilic molecules from spontaneously entering cells. This results in the situation that many compounds showing a promising potential in vitro cannot be applied in vivo because of delivery problems.
Many intracellular drug delivery systems were evaluated for their potential to transport therapeutic molecules inside cells. The genome sequencing and the huge knowledge resulting from that important technological revolution highly increased the interest to develop delivery tools.
Very important progresses were made which rise to many efficient transfection reagents allowing to study gene functions. However these nucleic acids intracellular delivery systems are still not adapted to in vivo development and only few therapeutic programs were developed.
In addition these systems which were developed for nucleic acids transfection and gene therapy are not adapted in most cases to the intracellular delivery of other biomolecules.
In addition, current technologies suffer from numerous limitations in terms of efficiency, toxicity, immunogenicity, cost and safety issues which do not allow exploiting the full potential of these potential therapeutic approaches. You will find below the main drug delivery systems developed for gene therapy.