17 December 2019

Accelerated evolution

Neuroscientists from Lund University in Sweden have developed a new technology for creating a viral envelope for targeted delivery of gene therapy only to target cells. The new technology was called BRAVE (Barcoded Rational AAV Vector Evolution). Researchers believe that it can be compared with a sharp acceleration of the process of evolution – from millions of years to several weeks.


A scheme for delivering genes in the viral envelope to dopamine-producing nerve cells that suffer from Parkinson's disease.

Some of the breakthrough therapies that have been introduced into clinical practice in recent years to treat complex diseases such as spinal muscular atrophy or enzyme deficiency are based on gene therapy.

In gene therapy, the genome of an organism is changed with the help of biological drugs. Examples of the approach are CRISPR/Cas9 gene scissors and CAR-T therapy.

For gene therapy, laboratory-grown delivery viruses are used, which are modified to be harmless and capable of delivering new genetic material to the cells of the body, replacing areas with damaged genes. The virus' own genome is completely removed.

The last five years of work by neuroscientist Thomas Bjerklund and his research team have led to the development of a mechanism that adapts these viral shells (capsids) so that they can accurately reach the type of cells in the body that need to be treated, for example, only nerve cells. The process combines powerful computer simulations and simulations with the latest gene and sequencing technologies.

Thanks to this work, it became possible to simultaneously study millions of new variants of viruses on cell cultures and animal models. As a result, the most suitable viral envelope is created for a specific application, in this case, dopamine–producing nerve cells for the treatment of Parkinson's disease.

Bjerklund writes about his discovery as a sharp acceleration of evolution from millions of years to weeks, because it allows you to study each "generation" of the virus in parallel with all the others in the same nerve cells and find out what makes a particular virus less effective. This is very important when creating computer models that interpret all the information.

The new method will help significantly reduce the need for laboratory animals, since millions of variants of one viral vector can be studied in the same individual. He will also transfer important stages of research from animals to human stem cell culture.

During the testing of the method, a new synthetic viral vector was created, which is suitable for gene therapy of Parkinson's disease and which can be used in clinical practice.

In collaboration with researchers from Harvard University, the group created a new biotech company, Dyno Therapeutics, in Boston to further develop virus engineering technology using artificial intelligence.

Article by M.Davidsson et al. A systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism is published in the journal PNAS.

Aminat Adzhieva, portal "Eternal Youth" http://vechnayamolodost.ru based on the materials of Lund University: High-tech method for uniquely targeted gene therapy developed.

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