BioCentury
ARTICLE | Translation in Brief

A telltale mouse to guide AAV design for gene editing

A mouse model puts in vivo AAV activity under the microscope and reveals unexpected opportunities for gene editing

August 22, 2019 11:14 PM UTC

A study from Spark co-founder Beverly Davidson suggests AAV vectors can reach more tissues than previously thought, opening up new opportunities and guidelines for AAV-mediated gene editing.

In a Nature Communications paper, Davidson’s team described a mouse model that reported transient, low levels of AAV transduction with more sensitivity than conventional methods. While such low levels of AAV activity are not expected to affect the performance of gene therapies, which require sustaining high levels of gene expression, the team showed they were sufficient to enable gene editing in unexpected tissues...