Toward personalized medicine in Bardet—Biedl syndrome

In this review from Personalized Medicine, find out how personalized medicine is being applied to treat rare ciliopathies.

Bardet—Biedl syndrome (BBS) is one of a family of disorders known as ciliopathies, all of which exhibit a defect in the structure or function of cilia. Advances in the understanding of ciliary biology and diagnostic techniques have opened up the prospect of treating BBS in a patient-specific manner. Promising avenues include gene therapy, gene editing techniques and splice-correcting and read-through therapies. Targeted drug design has been successful in the treatment of genetic disease. In this review from Personalized Medicine, find out more about research underway in the discovery of known and novel drugs to treat Bardet—Biedl syndrome.

Kenny J, Forsythe E, Beales P, Bacchelli C. Toward personalized medicine in Bardet—Biedl syndrome. Per. Med. 14(5), 447—456 (2017)