Abstract: Provided herein are methods for administering engineered B cells to produce a therapeutic agent in vivo. In various embodiments, engineered B cells are directly administered to the central nervous system (CNS). The compositions and methods disclosed herein may be used for enzyme replacement therapy, for example, treatment of diseases or disorders associated with lysosomal storage dysfunction through production of iduronidase (IDUA).

Abstract: Described are antibodies that specifically recognize and bind the epitope of glutamate decarboxylase (GAD65) that is bound by antibody b96.11, and anti-idiotypic antibodies that are capable of competing with GAD65 for binding with b96.11 and competitively inhibit such binding. These antibodies can be provided in the form of a pharmaceutical composition and can be used in methods for delaying the onset of Type 1 diabetes and for inhibiting insulitis and other diabetic symptoms. Also provided are methods for detecting a susceptibility to Type 1 diabetes in a subject and for detecting the presence of anti-idiotypic antibodies to GAD65. The method comprises contacting a specimen with an antibody of the invention. The method further comprises detecting binding of the molecule to the specimen. The absence (or relative absence) of binding is indicative of susceptibility to Type 1 diabetes and of the absence of anti-idiotypic antibodies.