Gene Therapies Near Or In Late-Stage Human Trials; FDA Approvals Probably Not Far Off

When Will Gene Therapy Come To The U.S.?  by Susan Young.  Technology Review.  September 30, 2013.

(1)  The Dutch biotech firm UniQure has been given approval by European regulators to sell its genetic treatment for ___________________.

(2)  Most gene therapies use a modified __________ to deliver a working copy of a gene to patients who lack a healthy version.