Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease.

At the highest dose employed, both vectors promoted a highly significant reduction in NAA relative to sham controls, with a 2.6-fold reduction in AAV/Olig001-ASPA brains (p = 5.06 × 10 −6 ) and a 2-fold reduction in AAV9-ASPA brains (p = 1.97 × 10 −6 ).

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Despite differences in relative transduction efficiency between the two serotypes, AAV/Olig001-ASPA brains manifest the greater reduction in NAA, but this reduction failed to reach statistical significance at any dose.

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