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Adeno-associated virus neutralising antibodies in type 1 diabetes mellitus

journal contribution
posted on 21.08.2019, 00:00 by S Andrzejewski, A Murali, Charmaine Ramlogan-SteelCharmaine Ramlogan-Steel, KP Edwards, N Efron, Jason SteelJason Steel, CJ Layton
Recombinant Adeno-associated viruses (AAVs) are an attractive vector for gene therapy delivery which may be blocked by AAV neutralising antibodies (NAbs). As Type 1 Diabetes (T1DM) is an endocrine disease of immunological origin, it is likely that NAb profiles are altered in the disease. In this study NAb to AAV2, AAV5, AAV6, and AAV8 in 72 subjects with T1DM and 45 non-diabetic patients were measured over a 4-year follow-up period. AAV2 NAb titres were significantly lower in non-diabetic subjects (P = 0.036). The T1DM group had more AAV8 NAb activity at baseline (P = 0.019), whilst after 4 years follow-up the T1DM group displayed developed increased AAV 5 (P = 0.03), 6 (P = 0.03) and 8 (P = 0.002) activity relative to the control group, however, overall AAV5 and 8 NAb levels were very low in patients <40. AAV NAb titre activity and prevalence generally appears higher in T1DM, however, low levels of AAV 5 and 8, particular in younger adult age groups at which T1DM can be targeted, could make these attractive vectors to target the disease.

Funding

Other

History

Volume

26

Issue

6

Start Page

250

End Page

263

Number of Pages

14

eISSN

1476-5462

ISSN

0969-7128

Location

England

Publisher

Springer Nature [academic journals on nature.com]

Language

eng

Peer Reviewed

Yes

Open Access

No

Acceptance Date

13/03/2019

External Author Affiliations

The University of Queensland; LVF Ophthalmology Research Centre, Qld; Queensland University of Technology

Era Eligible

Yes

Journal

Gene Therapy

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