Gene therapy has become safer

The risk of developing cancer associated with gene therapy has been reduced

Anna Stavina, XX2 century, based on WSU News: Safer gene therapy delivery reduces cancer risk

Scientists from Washington State University (Washington State University, WSU) have found a way to prevent the development of tumor cells – an infrequent but dangerous byproduct of gene therapy.

Grant Trobridge, associate professor of pharmacology, has changed the mechanism by which the virus delivers the desired gene to the target cell. An altered viral vector (the molecule used to deliver the gene) is associated with a reduced risk of cancer. The resulting vector can be used for the treatment of various hematological diseases.

Trobridge and his colleagues described their development in the journal Scientific Reports, an online publication published by the Nature publishing group (Hocum et al., Retargeted Foamy Virus Vectors Integrate Less Frequently Near Proto-oncogenes). The results of the group's work should form the basis of gene therapy with stem cells, which will be used to treat the deadly immunodeficiency of newborns SCID-X1, also known as "boy in the bubble syndrome".

Gene therapy is theoretically able to cure genetic diseases by replacing "broken" genes with "correct" copies. Clinical trials have shown impressive results, but at the same time, scientists have faced problems with the delivery of "new" genes and ensuring their normal operation for a long time. There were also questions about the safety of gene therapy. So, in an international study conducted in France and England, doctors managed to cure 17 out of 20 children with SCID-X1, only to immediately discover that five had developed leukemia.

A group of scientists led by Trobridge used a foamy retrovirus to create a viral vector (it got its name because of its ability to create foam in certain situations). Unlike other representatives of the retrovirus group, foamy retroviruses normally do not infect humans. In addition, they are less likely to activate nearby genes, including those associated with the development of cancer.

Retroviruses have become a natural basis for gene therapy, as they multiply by implanting their genes into the genome of the host organism.

Since the main task of the work was to create a safer viral vector, the scientists focused on how it would interact with the target stem cell. The virus had to be modified in such a way that it was embedded in safer parts of the genome and was less likely to be near genes that could cause cancer.

"Our goal is to create an effective and safe therapy for patients suffering from SCID–X1 and their families," said Trobridge. "We have already started collaborating with other scientists and practitioners."

According to the researcher, clinical trials of the new method will be conducted within the next five years.

Portal "Eternal youth" http://vechnayamolodost.ru 08.11.2016