Gene therapies are no doubt expensive and time-intensive. But their potential for immune deficiencies and blood-related disorders by using patient’s own stem cells are unique. More often, patients may require several therapies. Absence of viral vectors that can infiltrate through stems cells of interest accentuate the challenge. Most prominently, the failure is most evident when gene therapies involve hemopoietic stem cells.
Researchers have found that using specially engineered viruses, these cells can produce all types of blood cells. In gene therapy, usually small proportion of these stem cells act as sites for delivering gene therapies using a special set of viruses. These viruses comprise a set of lentiviral vectors. Their success or failure hinges on the fact that if these vectors can move past natural of proteins in hemopoietic stem cells.
Resveratrol-like Compound Key Enabler for Viral Vectors to Act Fast
Recently, what seems to be starting point, a team of scientists have hit success with resveratrol-like compound to make genetic modifications. The researchers they observed that resveratrol could act as key enabler for viral vector. Researchers at Scripps Center for Mass Spectrometry and Metabolomics Research Institution, California, have found when lentiviral vectors when combined with a compound they call caraphenol A, natural defense easily fall apart. Caraphenol A has properties similar to resveratrol.
The researchers performed the study on mice stem cells and found that the aforementioned technique makes the process markedly faster. Moreover, since the process is faster, the outcome is higher. The approach allows them to keep the self-renewing capacity of stem cells intact. The team found that the approach worked well for mice.
The researchers are looking further to get a better understanding of why stem cells resist genetic modification and what other possibilities exist. The focus is to improve gene therapies particularly for children.