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Recent advances in the development of gene delivery technologies

Published on February 23, 2022   40 min

A selection of talks on Pharmaceutical Sciences

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0:00
Hello to all. My name is Dr. Takis Athanasopoulos from GlaxoSmithKline. I'm the Acting Head of Patient Operations in Cell and Gene Therapy at GSK in Stevenage, UK. The focus of our talk today will be on the recent advances in the development of gene delivery technologies. It is an important goal in the research of gene delivery systems to develop clinically relevant vectors that we can use to combat a lot of diseases. We have quite a few HIV, AIDS, cancer, Alzheimer's and monogenic diseases. DNA or RNA-based viral vector systems utilise these viral or non-viral vector technologies to deliver genetic materials to host cells. It is an efficient way to deliver the genetic material and we will be capturing some of these recent technologies as part of our talk.
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Depicted here are some of the critical factors that are required for a successful cell and gene therapy. In terms of delivery, a viral or non-viral vector has lots of cell structures and defences to beat during their uptake. They have to utilise their cellular receptors, or co-receptors, but in order to work, it's trafficked towards the cell. There it will interact with multiple organelles, depending on whether it is a DNA or RNA vector. The eukaryotes include animals, plants and fungi, and we have multiple organelles with various functions. For example, the nucleolus that makes the ribosomes, the mitochondria which are the energy producers of the cell, the Golgi complex, an apparatus that processes proteins and lipids and are useful for the secretion of the cell, and microtubules that are for structural movement and cell division. The rough and the smooth endoplasmic reticulum for lipid synthesis and metabolism, and lysosomes and various other vesicles. Of importance to all these critical structures, and from a gene transfer perspective, is the uptake of the vector by appropriate cells, but also the subsequent steps, i.e., the vector entry into the nucleus if it is a DNA vector, or the cytoplasm if it is an RNA vector. Of paramount importance, from a gene transfer perspective, is the evasion of host immune defences. One of the factors required for successful cell and gene therapy transgene delivery is that the vector has to evade the host immune defences. These evasions are of multiple natures. It can be an innate response with subsequent release of pro-inflammatory cytokines, recruitment of neutrophils, macrophages, dendritic cells or other leukocytes, and activation and maturation of antigen-presenting cells. It can also involve adaptive priming through dendritic cell activation or adaptive responses. All of these are very important. From a cell and gene transfer delivery perspective, we want to abdicate or eliminate any unwanted immune responses.

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