The first diseases which benefited from gene therapy are rare genetic diseases in which the gene responsible had been identified, specifically severe immune deficiencies. (afm-telethon.com)
In June 2002, for example, a young girl treated with a new genetic therapy developed at the San Raffaele Hospital in Milan was cured of severe immune deficiency syndrome. (sciencemag.org)
The first diseases which benefited from gene therapy were rare genetic diseases in which the gene responsible had been identified, in particular severe immune deficiencies (afm-telethon.com)