A compound related to resveratrol could improve gene therapy delivery, potentially leading to improved treatment outcomes.
A research team at Scripps Research in La Jolla, CA, USA, say their findings could reduce the time and cost involved in current methods to deliver healthy genes into a patient’s blood stem cells.
Current gene therapy methods deliver therapeutic genes to haematopoietic stem cells via lentiviral vectors. These leverage the viruses’ natural capacity for inserting new genetic information into living cells.
However, haemopoietic stem cells are highly resilient to viral attacks, protecting themselves with interferon-induced transmembrane (IFITM) proteins which intercept lentiviral vectors. Therefore, it can take several attempts to successfully delivery genes into haemopoietic stem cells.
The study, led by Dr Bruce Torbett and published in the journal Blood, centres on caraphenol A, a small molecule closely related to the plant compound resveratrol found in grapes and red wine.
Torbett and his team focused on resveratrol and similar molecules to establish if they could enable viral vectors, used in gene therapy to deliver genes, to enter blood stem cells more easily.
He and his team found that adding caraphenol A to human haemopoietic stem cells caused the cells to let down their natural defences, reducing levels of IFITMs and relocating them to make them ineffective. This was confirmed when treated stem cells placed into mice divided and produced blood cells containing the new genetic information.
Dr Torbett says if gene delivery treatment of blood stem cells can be accomplished in less time, the cells can be re-administered to the patient sooner, which makes treatment more convenient for and also helps the stem cells to retain their self-renewing properties.
Ozog, S., Timberlake, N.D., Hermann, K., Garijo, O., Haworth, K.G., Shi, G., Glinkerman, C.M., Schefter, L.E., D'Souza, S., Simpson, E., Sghia-Hughes, G., Carillo, R.R., Boger, D.L., Kiem, H.P., Slukvin, I., Ryu, B.Y., Sorrentino, B.P., Adair, J.E., Snyder, S.A., Compton, A.A., Torbett, B.E. (2019) “Resveratrol trimer enhances gene delivery to hematopoietic stem cells by reducing antiviral restriction at endosomes”, Blood, doi: 10.1182/blood.2019000040
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