PNAs: computational biology helps to develop a unified targeted gene therapy

December 18, 2019 / BIOON / - recently, neuroscientists at Lund University in Sweden have developed a new technology that can transform the virus's shell to provide gene therapy with specific cell types In recent years, several new revolutionary therapies that have been used in bed to treat complex diseases (such as spinal muscular atrophy and enzyme deficiency) are based on gene therapy Through gene therapy, biological drugs can be used to control or change genetic material For example, CRISPR / cas9 gene editing system and so-called car-t cells used to treat various forms of cancer (image source: www Pixabay Com) in the past five years, neuroscientist tomasbj ö rklund and his team have developed a process of customizing the viral capsid so that they can precisely reach cells in the body that need treatment, such as neurons This process involves computer modeling and the combination of gene technology and sequencing technology "With this technology, we can study millions of new virus variants in both cell culture and animal models After that, we can use computer simulation to select the most appropriate virus shell and target dopamine secreting neurons to treat Parkinson's disease, "said Tomas BJ ö rklund, senior lecturer in translational neuroscience at Lund University Using this new method, researchers can greatly reduce the demand for experimental animals, and they can also transfer research from animals to in vitro cultures of human stem cells "We believe that the new synthetic virus we have successfully created is very suitable for gene therapy of Parkinson's disease, and we very much hope that these virus vectors can be put into clinical use We have set up a new biotech company, dyno therapeutics, in Boston to further exploit artificial intelligence to develop virus engineering technology for future treatment " Tomasbj ö rklund concluded Information source: high tech method for uniquely targeted gene therapy developed original source: Marcus Davidson, Gang Wang, Patrick Aldrin Kirk, Tiago Cardoso, Sara nolbrant, Morgan hartnor, janitha mudanayake, Malin Parmar, Tomas Björklund A systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism Proceedings of the National Academy of Sciences , 2019; 201910061 DOI: 10.1073/pnas.1910061116