Sickle cell disease is a genetic illness that modifies the shape of red blood cells into a crescent or sickle shape, causing serious health issues and heightening the risk of patients developing blood cancer. A recently conducted study has found promising results concerning gene therapy’s potential in curing genetic illnesses. These findings providing new insights into what happens in the human body following treatment.
For their study, the researchers examined samples obtained from sickle cell disease patients who underwent gene therapy as part of a trial conducted by Boston Children’s Hospital. They then observed the genetic changes in each patient’s stem cells, before and after they underwent treatment. Once this was done, they compared the cells the treatment altered to those that remained the same, using new genome science technologies.
Dr. David Williams, cosenior author of the study, stated that gene therapy had the potential to cure genetic illnesses such as sickle cell disease, noting that understanding how the treatment influenced the growth of blood stem cells was essential for safety, especially in the long-term. Williams added that their research had shown that younger patients who had fewer genetic mutations in their stem cells didn’t demonstrate strong signs of mutations after their treatments. This, he continued, suggested that treating patients with gene therapy from a young age could be more effective as well as safer.
Professor David Kent, the study’s cosenior author, explained that their research had shown that gene therapy could create an environment that supported growth of stem cells with specific mutations, which could possibly cause blood cells that had these mutations to increase. In some settings, he continued, such increases could be linked to blood-cancer development, especially in older patients.
However, he noted, the link between the research’s findings and the risk of developing blood cancers hadn’t been explored fully. This research underscores the importance of in-depth and long-term monitoring of samples of stem cells from patients with genetic illnesses.
It should be noted that the study’s findings show that while gene therapies aren’t the likely cause of new mutations in stem cells, genetically modifying these stem cells and reintroducing them to a patient’s body does make mutations more prominent, which in turn increases their influence on the blood and immune systems.
The study’s co-lead authors include Dr. Alyssa Cull and Michael Spencer-Chapman of the Wellcome Sanger Institute. The study findings were reported in “Nature Medicine.” It was funded by the Bill and Melinda Gates Foundation.
As more companies such as Renovaro BioSciences Inc. (NASDAQ: RENB) venture into developing gene therapies for various diseases, mechanisms are likely to be found to improve the long-term safety profiles of these treatments so that costly follow-up tests are minimized.
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