News of Note—Stabilizing viral vectors for gene therapy; a nonantibiotic approach to resistant infections

DNA genomics precision medicine
Penn researchers reported that the viral vectors used to deliver gene therapy can mutate during manufacturing and that they may have hit on a way to prevent the problem. (Pixabay)

Improving gene therapy with more efficient viral vectors
The viral vectors that are used to transport therapeutic genes into the body can undergo unexpected changes during manufacturing that cause them to lose functioning, scientists at the University of Pennsylvania’s Perelman School of Medicine have discovered. The adeno-associated virus that’s commonly used in gene therapy consists of 60 interlocking proteins—any of which could spontaneously mutate during manufacturing. So the Penn team developed a new process that stabilizes the protein shell to improve its functionality. The process, described in the journal Molecular Therapy, could facilitate the development of lower doses of gene therapy, which could in turn reduce side effects, the researchers believe. (Release)

Treating drug-resistant bacteria without antibiotics
Researchers at Case Western Reserve University are investigating a new method for treating resistant bacterial infections with small molecules that bind to and cripple a toxin-creating protein in gram-positive bacteria called AgrA. The compounds rendered the bacteria ineffective, while also boosting the ability of antibiotics to do their job, the team reported in the journal Scientific Reports. In mouse models of methicillin-resistant Staphylococcus aureus, the compounds produced 100% survival. When they were combined with antibiotics, the animals had 10-times-fewer bacteria in their bloodstream than what was achieved with antibiotics alone, they said. Two of the compounds have been licensed to Israel-based Q2Pharma for further development. (Release)

An RNA that may suppress breast cancer metastasis
Scientists at the University of Texas MD Anderson Cancer Center have discovered that a type of RNA called metastasis-associated lung adenocarcinoma transcript 1 (MALAT1) suppresses the spread of breast cancer in mouse models. They were surprised by the finding, because MALAT1 was previously reported to be a promoter of cancer metastasis. But the team found that MALAT1 disrupts a protein complex that causes cancer cells to proliferate and spread. In a mouse model of breast cancer, inactivating the MALAT1 gene promoted lung metastasis, the researchers reported in the journal Nature Genetics. They were able to reverse the spread by reintroducing the RNA. (Release)


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