Cellular gene therapy

Experiments on a model of a severe hereditary disease have shown the possibility of using cell therapy for genetic correction

Marina Astvatsaturyan, Echo of Moscow

In the work carried out by Harvard Medical School scientists under the Howard Hughes Medical Institute program, the same stem cells are used, the prospects of which in the restoration of affected organs and tissues are now being discussed everywhere.

Stem cells are plastic immature material, thanks to most of it, the embryo grows into an adult organism, the remaining negligibly small amount is a cellular depot scattered throughout the body, cells capable of giving rise to healthy tissues under certain conditions, they are even in the brain.

But the main task of researchers led by Dr. Emanuela Guzzoni is the genetic correction of a hereditary defect, an ailment called Duchene's myodystrophy, a disease of underdevelopment of all muscles caused by a mutation of a single gene.

Gene therapy today is an attempt to introduce into the patient's body a genetically engineered design based on a virus, although neutralized, but theoretically capable of turning into a danger.

The results obtained in Guzzoni's laboratory allow us to hope that instead of a virus, a stem cell – the precursor of a future normal muscle cell - can be used as a gene carrier. This method, unlike a gene-therapeutic injection exactly into the muscle, will ensure the delivery of the "right" gene to the place where the cellular treatment will take place. The work was published in the latest issue of the journal Nature.

The experiment used a special line of mice that do not produce the protein dystrophin – this is a model of the disease known in humans as Duchenne muscular dystrophy.

Females of such mutant animals, having received a dose of radiation, were deprived of their own hematopoietic stem cells, the source of which is bone marrow, then they were injected with stem cells isolated from normal males. The success of such a transfer by itself can be assessed immediately, since the chromosomal set of females and males is different, but the main thing is that the imported material carries the normal protein dystrophin and its gene, which, to the surprise of scientists, began to work in sick females, ensuring the development of normal muscle cells of model animals.

These are the preliminary results, and in parallel with their publication, one of the authors of the work, Richard Mulligan, raises the question that has already been raised about the therapeutic potential and origin of stem cells, because it turns out that normal muscle cells develop from the precursors of hematopoietic cells.

Portal "Eternal youth" http://vechnayamolodost.ru  27.09.2016