A novel approach to quantitate biodistribution and transduction of adeno-associated virus gene therapy using radiolabeled AAV vectors in mice.

ABSTRACT: An understanding of recombinant adeno-associated virus (AAV) biodistribution profiles is an important element of a preclinical development program. Here, we have developed a radiolabeling strategy utilizing the co-delivery of ¹²⁵I (non-residualizing) and ¹¹¹In (residualizing) radionuclide-conjugated AAVs to provide a detailed distribution quantification at tissue level delineating between the cellular internalized AAV (degraded, ¹¹¹In-¹²⁵I) and AAV remaining in the extracellular matrix (intact, ¹²⁵I). This labeling method has been successfully applied to AAV9 and AAV-PHP.eB as tool molecules without altering the physical properties and biological activities of the AAVs. Upon labeling with either of the radioactive probes, these molecul

SUBMITTER: Wang H 

PROVIDER: S-EPMC11404148 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature