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Preclinical Assessment in Transgenic NOD Mice of a Novel Immunotherapy for Type 1 Diabetes: Lipoplexes down Modulate the Murine C1858T <em>Ptpn22</em> Variant <em>In </em><em>Vitro</em>

2025-10-11

Abstract excerpt

The C1858T PTPN22 (R620W) variant has been implicated in the pathogenesis of several autoimmune disorders and represents a promising immunotherapeutic target for Type 1 diabetes. We have been implementing a novel immunotherapeutic approach based on the use of lipoplexes that deliver siRNA duplexes. Efficacy and safety of lipoplexes halting variant expression was demonstrated in the peripheral blood of patients in...

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Literature Corpus work
0cc0e875-5bf6-5a86-9747-38bf6624d30c
DOI
10.20944/preprints202510.0849.v1
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Preclinical Assessment in Transgenic NOD Mice of a Novel Immunotherapy for Type 1 Diabetes: Lipoplexes down Modulate the Murine C1858T <em>Ptpn22</em> Variant <em>In </em><em>Vitro</em>DOI 10.20944/preprints202510.0849.v1
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