New preprint! We identify the precise origin of DNA Rep/Cap contaminants previously observed in an AAV patient liver biopsy to be the result of an AAV P5 promoter placed directly after the AAV capsid gene in the manufacturing design.
Great collaboration with UCL to build upon their prior findings.
medrxiv.org
P5 promoter-mediated incorporation explains REP/CAP manufacturing contaminants in patient liver after rAAV gene therapy
Sequencing of liver tissue from a patient treated with the rAAV gene therapy Zolgensma for spinal muscular atrophy recently revealed contaminating plasmid sequences derived from rAAV manufacturing wit...