2024
DOI: 10.1111/jcmm.18460
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AAV mediated repression of Neat1 lncRNA combined with F8 gene augmentation mitigates pathological mediators of joint disease in haemophilia

Pratiksha Sarangi,
Mohankumar B. Senthilkumar,
Sonal Amit
et al.

Abstract: Haemophilic arthropathy (HA), a common comorbidity in haemophilic patients leads to joint pain, deformity and reduced quality of life. We have recently demonstrated that a long non‐coding RNA, Neat1 as a primary regulator of matrix metalloproteinase (MMP) 3 and MMP13 activity, and its induction in the target joint has a deteriorating effect on articular cartilage. In the present study, we administered an Adeno‐associated virus (AAV) 5 vector carrying an short hairpin (sh)RNA to Neat1 via intra‐articular inject… Show more

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