Delivering efficient liver-directed AAV-mediated gene therapy
Author:
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
https://www.nature.com/articles/gt201690.pdf
Reference18 articles.
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3. Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014; 371: 1994–2004.
4. D'Avola D, Lopez-Franco E, Sangro B, Paneda A, Grossios N, Gil-Farina I et al. Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria. J Hepatol 2016; 65: 776–783.
5. Paneda A, Lopez-Franco E, Kaeppel C, Unzu C, Gil-Royo AG, D'Avola D et al. Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria. Hum Gene Ther 2013; 24: 1007–1017.
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