Source 1primary paper2026MED
Toolkit/AAV9-DM
AAV9-DM
Taxonomy: Mechanism Branch / Architecture. Workflows sit above the mechanism and technique branches rather than replacing them.
Summary
Here we utilized rational design techniques to make five point mutations in the AAV9 capsid. In doing so, we developed a novel AAV9 variant, AAV9-DM, that is characterized by reduced liver tropism as compared to AAV9 and other liver de-targeted AAV9 mutants.
Usefulness & Problems
No literature-backed usefulness or problem-fit explainer has been materialized for this record yet.
Published Workflows
Objective: Develop a safer AAV9-based vector for systemic targeting of extrahepatic tissue by reducing liver targeting while preserving non-hepatic transduction.
Why it works: The abstract frames liver detargeting as a crucial first step toward safer systemic AAV9 delivery, then describes rational capsid mutation followed by in vivo testing to identify a variant that reduces liver tropism while retaining extrahepatic distribution.
altered capsid tropism through five point mutations in the AAV9 capsidrational designcomparative in vivo evaluation
Stages
- 1.Rational capsid design(library_design)
The abstract states that reducing liver targeting is a crucial first step toward a safer AAV9-based vector, motivating rational design of capsid mutations.
Selection: Introduce five point mutations into the AAV9 capsid to reduce liver targeting.
- 2.Comparative in vivo tropism and expression evaluation(confirmatory_validation)
The engineered capsid must be shown to remain effective in vivo while reducing liver targeting.
Selection: Assess reduced liver tropism, preserved non-hepatic biodistribution, and sustained transgene expression relative to AAV9 and other liver de-targeted mutants.
Taxonomy & Function
Primary hierarchy
Mechanism Branch
Architecture: A delivery strategy grouped with the mechanism branch because it determines how a system is instantiated and deployed in context.
Mechanisms
capsid tropism retargetingTechniques
Computational DesignTarget processes
No target processes tagged yet.
Validation
Cell-freeBacteriaMammalianMouseHumanTherapeuticIndep. Replication
Supporting Sources
Ranked Claims
AAV9-DM maintains the ability to transduce non-hepatic tissues with a biodistribution similar to AAV9.
Rational design of five point mutations in the AAV9 capsid produced the novel variant AAV9-DM.
AAV9-DM effectively transduces cells in vivo and produces robust transgene expression over a 9-week period.
transgene expression duration 9 week
AAV9-DM may provide a basis for safer therapeutics targeting extrahepatic tissue while reducing adverse side effects related to liver transduction.
AAV9-DM has reduced liver tropism compared with AAV9 and other liver de-targeted AAV9 mutants.
Approval Evidence
1 source5 linked approval claimsfirst-pass slug aav9-dm
Here we utilized rational design techniques to make five point mutations in the AAV9 capsid. In doing so, we developed a novel AAV9 variant, AAV9-DM, that is characterized by reduced liver tropism as compared to AAV9 and other liver de-targeted AAV9 mutants.
Source:
biodistributionsupports
AAV9-DM maintains the ability to transduce non-hepatic tissues with a biodistribution similar to AAV9.
Source:
engineering outcomesupports
Rational design of five point mutations in the AAV9 capsid produced the novel variant AAV9-DM.
Source:
in vivo transductionsupports
AAV9-DM effectively transduces cells in vivo and produces robust transgene expression over a 9-week period.
Source:
therapeutic relevancesupports
AAV9-DM may provide a basis for safer therapeutics targeting extrahepatic tissue while reducing adverse side effects related to liver transduction.
Source:
tropism changesupports
AAV9-DM has reduced liver tropism compared with AAV9 and other liver de-targeted AAV9 mutants.
Source:
Comparisons
No literature-backed comparison notes have been materialized for this record yet.
Ranked Citations
- 1.