In a previous article, we analyzed the contours of the common law experimental use exemption. Now, we will explore the ...

Mass photometry enables rapid, purification-free quantification of rAAV vectors, offering a faster solution for gene therapy ...

The gene editor -- called evoCAST -- goes a long way toward solving a problem that has confounded the development of gene therapies from the field's beginnings: How to add long stretches of DNA to ...

Iterations of AAVPureMfg are developed with new plasmid structure design and reduced plasmid demand. AAVPureMfg substantially improves rAAV product purity by mitigating prokaryotic DNA encapsidation ...

Many, but not all, fibrillin-1 gene mutations are expected to exert a dominant negative effect, whereby mutant fibrillin monomers impair the global function of the microfibrils. In this paper we ...