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Reduce the risk of liver damage and other serious side effects
When Sharif Tabebordbar was a teenager, his father began to have difficulty walking
"I watch my dad get worse day by day," Tabebordbar said
Tabebordbar is now a scientist at the Broad Institute of MIT and Harvard University, and an assistant researcher in the Department of Organisms and Evolutionary Biology.
Together with colleagues at Broad and Harvard University, Tabebordbar has taken another step toward this goal with a new gene delivery system that has the potential to make gene therapy for muscle diseases safer and more effective for patients
The system is called MyoAAV and is published in Cell magazine
It is a new family of adeno-associated viruses, a better vector for gene therapy, and can be used to carry the gene editing system sometimes called CRISPR 2.
In this study, the researchers showed that the group of viral vectors they created can reach and deliver therapeutic muscle cells more effectively, 10 times that of other gland-related vectors, and reach about 100 to 250 times at lower doses
The system has achieved some impressive results
"All these results demonstrate the broad applicability of MyoAAV vectors for delivery to muscles," said Amy Wagers, co-senior author of the study.
This paper details how the team modified the outer protein coat of the adeno-associated virus capsid AAV9, which is a commonly used gene delivery vector in gene therapy to improve its ability to deliver genes to muscle cells
Then, treat inherited muscle diseases in animal models
In collaboration with the Alan Beggs laboratory of Boston Children's Hospital, the research team found that MyoAAV is also effective in treating Duchenne muscular dystrophy
For the mouse model of x-linked myotubular myopathy, the researchers used a dose 100 times lower than other viral vectors currently used in clinical trials, and found that all six MyoAAV mice in the study had the same life span as normal mice.
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In contrast, mice treated with AAV9 can only live to 21 weeks of age
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In their final experiment, the research team found that MyoAAV, which is designed for non-human primates, delivers genes to the muscles of these animals and is much more effective than the naturally occurring capsids used in current clinical trials
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MyoAAV has also successfully introduced genes into human cells in the laboratory
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The results indicate that MyoAAV can be used for muscle-directed gene delivery between different species, because different MyoAAV capsids use similar mechanisms to deliver genes to mouse and human muscle cells
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Next, we will further use the data from the research to study how to use the system to develop effective drugs for patients
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"We have a lot of information about this type of carrier, and many exciting new studies can be initiated from this information," Wagers said
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Original title:
Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species
