What’s next in gene therapy for Crigler-Najjar syndrome?

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Declaration of interest

G Ronzitti is author of patents related to the development of gene therapies for rare metabolic diseases and in particular, of one patent regarding AAV gene therapy for CN syndrome. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

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Funding

This paper was not funded.