Engineered capsids for efficient gene delivery to the eye

A rational design approach created novel variants of adeno-associated viral (AAV) capsids. These have improved transduction properties in the mouse retina and cornea. as reported in the peer-reviewed journal Human Gene Therapy.

The efficient gene delivery of these variants was confirmed in non-human primate tissue. “The capsid modified AAV2 and AAV5 variants described here have novel attributes that will add to the efficacy and specificity of their potential use in gene therapy for a range of human ocular diseases,” said Catherine O’Riordan and coauthors from Sanofi.

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