Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
Author:
Funder
Action Medical Research
Great Ormond Street Hospital Charity
Medical Research Council
Publisher
Wiley
Subject
Genetics(clinical),Genetics
Link
http://link.springer.com/article/10.1007/s10545-017-0053-3/fulltext.html
Reference238 articles.
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3. Angelis A, Tordrup D, Kanavos P (2015) Socio-economic burden of rare diseases: a systematic review of cost of illness evidence. Health Policy 119:964–979
4. Annoni A, Goudy K, Akbarpour M, Naldini L, Roncarolo MG (2013) Immune responses in liver-directed lentiviral gene therapy. Transl Res 161:230–240
5. Apolonia L, Waddington SN, Fernandes C et al (2007) Stable gene transfer to muscle using non-integrating lentiviral vectors. Mol Ther 15:1947–1954
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