A single epidermal stem cell strategy for safe ex vivo gene therapy.

ABSTRACT: There is a widespread agreement from patient and professional organisations alike that the safety of stem cell therapeutics is of paramount importance, particularly for ex vivo autologous gene therapy. Yet current technology makes it difficult to thoroughly evaluate the behaviour of genetically corrected stem cells before they are transplanted. To address this, we have developed a strategy that permits transplantation of a clonal population of genetically corrected autologous stem cells that meet stringent selection criteria and the principle of precaution. As a proof of concept, we have stably transduced epidermal stem cells (holoclones) obtained from a patient suffering from recessive dystrophic epidermolysis bullosa. Holoclones were infected with self-inactivating retroviruses bearing a

SUBMITTER: Droz-Georget Lathion S 

PROVIDER: S-EPMC4403041 | biostudies-literature | 2015 Apr

REPOSITORIES: biostudies-literature