Intra-CNS AAV9-GBA1 delivery yields species and route of administration differences in safety and transgene expression.

Ana Claudia Amaral¹, Branka Grubor¹, Davide Gianni¹, Lee Koetzner¹, Neeta Abraham¹, Shawn Bourque¹, Danielle Brown², Yi Chen¹, Kay-Ellen Chicoine¹, Pete Clarner¹, P J De Giovanni¹, Stefan Hamann¹, Melissa Kirkland¹, Odete R Mendes², Merilla Michael¹, Murali V P Nadella¹, Kalyani Nambiar¹, Jennifer Sebalusky¹, Weike Zeng¹, Shanqin Xu¹, Patrick Trapa¹, Edward D Plowey¹, Eric Tien¹, Jim Fikes¹, Dominic M Walsh¹, Warren D Hirst¹, Junghae Suh¹, Kelly E Glajch¹

¹Biogen, Cambridge, MA 02142, USA.

²Charles River Laboratories, Durham, NC 27709, USA.

PubMed PMC DOI PDF

Abstract

Adeno-associated virus (AAV) delivery of lysosomal enzymes to the brain is currently being tested in several clinical trials. However, publicly available non-human primate (NHP) studies comprehensively reporting biodistribution, transgene expression, and safety across the central nervous system (CNS) remain limited. Here, we examined AAV9-GBA1 (encoding glucocerebrosidase [GCase]) delivery to mice…

Create a free account to keep reading

Free members get 10 full research views every month across publications, clinical trials, FDA clearances, adverse events, and NIH grants. No credit card required.

Create Free Account Sign In

Want unlimited research access? See Pro plans

Data Accuracy Notice: Research intelligence on Health AI Central is aggregated from public sources (PubMed, ClinicalTrials.gov, FDA, NIH, CMS, and others) and refreshed nightly. Classifications and derived metrics are produced by automated methods described in our Methodology. We recommend verifying critical data points against the primary sources before making decisions.