Scientists from Germany have proposed a method of performing gene therapy directly inside the body, without the preliminary work with the defective cells in the laboratory. This approach can cure not only genetic diseases, but also cancer.
Scientists from Halle-Wittenberg University have developed a new means of DNA delivery for gene therapy. Their method uses artificial lipids and not modified viruses, which are now actively being investigated for the treatment of congenital immunodeficiency, blindness, sickle cell anemia, cancer and other serious diseases.
This method has its drawbacks. Modification of the virus for delivery of genetic material is an expensive method, requires a long preparation but most importantly — is associated with risk of strong immune response. To solve these problems, scientists began to study liposome — they already use to deliver to the body of other drugs.
Liposomes combine with nucleic acids to DNA to form so-called lipoplexes, explain the authors. Then merge with the cell membrane and release genetic material into the content of the cell. Scientists have developed four synthetic lipid, which effectively transported DNA. They can encapsulate nucleic acids, protect them from enzymatic degradation and enter into the cells.
One of the four lipids called DiTT4 scientists will investigate in the preclinical stages on animal models, however, have yet to find out the specifics of the release of genetic material when DiTT4 will be entered directly into the body.
“DiTT4 so compatible with blood components that will allow us to perform in the future gene therapy within the body,” said the authors.
If so, then technology will mark the beginning of a revolution in genetic medicine: current methods of gene therapy require the modification of cells in vitro and their subsequent administration.
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