Intrathecal sc-AAV9-CB-GFP: Systemic Distribution Predominates Following Single-Dose Administration in Cynomolgus Macaques.

ABSTRACT: Biodistribution of self-complementary adeno-associated virus-9 (scAAV9)-chicken β-actin promoter-green fluorescent protein (GFP) was assessed in juvenile cynomolgus macaques infused intrathecally via lumbar puncture or the intracisterna magna (1.0×10¹³ or 3.0×10¹³ vg/animal), with necropsy 28 days later. Our results characterized central nervous system biodistribution compared with systemic organs/tissues by droplet digital polymerase chain reaction for DNA and in situ hybridization. Green fluorescent protein expression was characterized by Meso Scale Discovery electrochemiluminescence immunosorbent assay and immunohistochemistry (IHC). Biodistribution was widespread but variable, with vector DNA and GFP expression greatest in the spinal cord, dorsal root gangl

SUBMITTER: Meseck EK 

PROVIDER: S-EPMC9284083 | biostudies-literature | 2022 Jun

REPOSITORIES: biostudies-literature