6533b856fe1ef96bd12b1e5b

RESEARCH PRODUCT

Gene therapy for type 1 diabetes: is it ready for the clinic?

Pleunie P. M. RoodPleunie P. M. RoodNick GiannoukakisNick GiannoukakisAntonella D’anneoA.n. BalamuruganA.n. BalamuruganJing HeJing HeRita BottinoRita Bottino

subject

Diabetes; Gene therapy; Immunotherapy; Autoimmunity.medicine.medical_treatmentGenetic enhancementTransgeneImmunologyGenetic VectorsAutoimmunity.BioinformaticsDiabeteAdenoviridaeGene therapyAntigenmedicineAnimalsHumansProgenitor cellgeographyType 1 diabetesgeography.geographical_feature_categorybusiness.industryInsulinGene Transfer TechniquesImmunosuppressionGenetic TherapyIsletmedicine.diseaseDiabetes Mellitus Type 1Immunotherapybusiness

description

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.

10.1385/ir:36:1:83http://hdl.handle.net/10447/25215