The Critical Role of First-Principles, Fundamental Techniques in Gene Therapy


Date
Mar 28, 2023 12:00 AM
Event
Mar 2023 | Gene Therapy Comparability Summit (Invited)
Sep 2022 | Viral Vector Characterization Summit (Invited)
Oct 2023 | Beckman AUC User’s Group Meeting (Invited)

All current methods used to characterize AAV [empty/partial/full] are compared and contrasted along with several case studies highlighting the utility of first-principles, fundamental techniques such as UV-Vis, DLS, and SV-AUC in gene therapy characterization. These methods are useful for deconvoluting interpretation errors caused by ‘Jingle’ fallacies common in gene therapy analytics - e.g., the difference between mass and number weightings when considering constructs such as “%empty” and “%HMW”. While it’s tempting to neglect these methods once more routine, abstracted methods such as PCR are online, abstracted are at higher risk for method induced artifacts.

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