High-throughput evaluation of cardiac-specific promoters for adeno-associated virus mediated cardiac gene therapy

Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, et al. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 2010;363:355–64.

CAS 
PubMed 
PubMed Central 

Google Scholar 

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.

PubMed 
PubMed Central 

Google Scholar