Research graph
References from Advancing AAV technology—From promise to product. Local targets link to admitted publications; unresolved targets remain external evidence.
Identification of a novel neutralization epitope in rhesus AAVs
10.1016/j.omtm.2024.101350 · 2024 · External reference
The combination of rAAV pseudo-lipid nanoparticle and triamcinolone acetonide enables multi-administration to liver
10.1016/j.omtm.2024.101399 · 2025 · External reference
AAV2 delivery of the saCas9 gene results in presentation of an HLA-A( ∗)02:01-restricted T cell epitope potent to induce T cell cytotoxicity
10.1016/j.omtm.2025.101506 · 2025 · External reference
DNA-PK inhibition enhances gene editing efficiency in HSPCs for CRISPR-based treatment of X-linked hyper IgM syndrome
10.1016/j.omtm.2024.101297 · 2024 · External reference
Comprehensive analysis of off-target and on-target effects resulting from liver-directed CRISPR-Cas9-mediated gene targeting with AAV vectors
10.1016/j.omtm.2024.101365 · 2024 · External reference
Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I
10.1016/j.omtm.2024.101369 · 2024 · External reference
Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I
10.1016/j.omtm.2024.101363 · 2024 · External reference
Canine and Feline Models of Human Genetic Diseases and Their Contributions to Advancing Clinical Therapies
2017 · External reference
Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus
10.1016/j.omtm.2024.101263 · 2024 · External reference
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies
10.1016/j.omtm.2024.101273 · 2024 · External reference
