Which gene therapy approach will prove most safe and effective for clinical PKU treatment?

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Priority

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Importance

82%

Tractability

70%

Market price

50%

Description

Multiple gene therapy methods are being tested (viral vectors, non-viral delivery, gene editing) but no clear winner has emerged from preclinical studies. This uncertainty hampers clinical translation and resource allocation for PKU neurodegeneration prevention.

Gap type: open_question Source paper: State-of-the-art 2023 on gene therapy for phenylketonuria. (None, None, PMID:37401651)

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