AAV-mediated liver-directed gene therapy for Acute Intermittent Porphyria: It is safe but is it effective?
Author:
Funder
European Research Council
Fondazione Telethon
National Institute for Health Research
NIHR
Publisher
Elsevier BV
Subject
Hepatology
Reference17 articles.
1. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B;Nathwani;N Engl J Med,2011
2. Long-term safety and efficacy of factor IX gene therapy in hemophilia B;Nathwani;N Engl J Med,2014
3. Gene therapy enters the pharma market: the short story of a long journey;Buning;EMBO Mol Med,2013
4. Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria;D’Avola;J Hepatol,2016
5. Acute porphyrias in the USA: features of 108 subjects from porphyrias consortium;Bonkovsky;Am J Med,2014
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2. Multiple roles of haem in cystathionine β-synthase activity: implications for hemin and other therapies of acute hepatic porphyria;Bioscience Reports;2021-07
3. Acute Intermittent Porphyria: An Overview of Therapy Developments and Future Perspectives Focusing on Stabilisation of HMBS and Proteostasis Regulators;International Journal of Molecular Sciences;2021-01-12
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5. A Pharmacological Chaperone Therapy for Acute Intermittent Porphyria;Molecular Therapy;2020-02
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