title: Gene therapy for type 1 diabetes: is it ready for the clinic?
abstract: This review, in addition to updating the growing list of type 1 diabetes- relevant gene therapies, offers an outline of short-term objectives that can readily be met to move, at least, adenoviral and adeno-associated viral-based protocols into the clinic, first as a means of facilitating islet allografts as well as platforms with which to introduce immunoregulatory transgenes. A wide array of genes have been tested to restore insulin production, to drive the differentiation of insulin-producing progenitors, and to confer immunosuppression in an antigen- and tissue-restricted manner.
authors.affiliation: Diabetes Institute, Children's Hospital of Pittsburgh, Pittsburgh 15213, USA.
authors.forename: Antonella
authors.initials: A
authors.lastname: D'Anneo
authors.forename: Pleunie
authors.initials: P
authors.lastname: Rood
authors.forename: Rita
authors.initials: R
authors.lastname: Bottino
authors.forename: A N
authors.initials: AN
authors.lastname: Balamurugan
authors.forename: Jing
authors.initials: J
authors.lastname: He
authors.forename: Nick
authors.initials: N
authors.lastname: Giannoukakis
mesh_terms: D000256:Adenoviridae / Q000235:genetics; D000818:Animals; D003922:Diabetes Mellitus, Type 1 / Q000235:genetics / Q000628:therapy*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans