Our study provides insight into state-of-the-art translational and drug-development considerations for engineered neurotropic and ocular capsids.
First-pass extracted concept
engineered ocular and neurotropic AAV capsids
Candidate: concept label1 source documents3 linked claims
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Evidence Snippets
Supporting Sources
Linked Claims
Engineered capsids in the reviewed set exhibit enhanced tissue specificity, improved pharmacokinetics and pharmacodynamics, or reduced off-target effects compared with parent serotypes.
Engineered neurotropic and ocular AAV capsids are being developed to enhance tissue and cell-specific targeting compared with naturally occurring serotypes.
Natural AAV serotypes often lack specificity and efficiency, contributing to off-target effects and a low therapeutic index.