Gene therapy for type 1 diabetes: is it ready for the clinic?
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.
D'Anneo
Antonella
A
Diabetes Institute, Children's Hospital of Pittsburgh, Pittsburgh 15213, USA.
Rood
Pleunie
P
Bottino
Rita
R
Balamurugan
A N
AN
He
Jing
J
Giannoukakis
Nick
N
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