Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

Baruteau, J; Waddington, SN; Alexander, IE; Gissen, P; (2017) Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. Journal of Inherited Metabolic Disease 10.1007/s10545-017-0053-3 . (In press). Green open access

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