HUMAN GENE THERAPY 22:1–4 (June 2011),DOI: 10.1089/hum.2011.2504
"Research and outcomes like these give hope not only to patients, but to doctors who have spent years developing gene therapies for diseases that have not responded as well to traditional therapy.The promise shown by the results also create hope that gene therapy can finally emerge as avibrant segment, after years in which biopharma companies, physicians, insurers, and government regulators have struggled to develop a sustainable business model."
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