Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins

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Population

Mice, including hemophiliac, factor IX-deficient mice

Comparison

Intranasal delivery of AAV vectors encoding… vs Comparison between different AAV capsids

Design

Preclinical

Follow-up

Up to 150 days (5 months)

Authors

Discussion

Intranasal AAV5 supports further preclinical gene therapy development; leaves open safety, efficacy, and translation to humans.

Population

Mice, including hemophiliac, factor IX-deficient mice

Intervention

Intranasal delivery of AAV vectors (AAV2/1, AAV2/2, or AAV2/5) encoding cellular markers, erythropoietin (Epo), or factor IX

Comparator

Comparison between different AAV capsids (AAV1, 2, or 5)

Outcome

Gene expression levels in airways and alveoli, and serum levels of secreted therapeutic proteins (Epo, factor IX)surrogate

Intranasal administration of AAV5 vectors enables efficient gene transfer to the lung and sustained systemic delivery of therapeutic proteins.

Auricchio et al. (2002) studied this question.

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