Case Report: Telitacicept in treating a patient with NF155+ autoimmune nodopathy: a successful attempt to manage recurrent elevated sero-anti-NF155 antibodies

Ren, Yijun; Chen, Si; Yang, Huan

doi:10.3389/fimmu.2023.1279808

Cited by 4 publications

(1 citation statement)

References 22 publications

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“…Telitacicept has been granted orphan drug status of MG by the Food and Drug Administration (FDA) and received its first approval for treating SLE in China [ 36 ]. Our previous research has also reported successful therapy outcomes of telitacicept on neurofascin-155 (NF155) + autoimmune nodopathy [ 37 ]. Further clinical trials of telitacicept on MG are necessary, and a phase 3 clinical trial of telitacicept for generalized AChR + MG therapy is being conducted by our research group.…”

Section: Discussionmentioning

confidence: 99%

Mendelian randomization and colocalization analysis reveal novel drug targets for myasthenia gravis

Ouyang,

Chen,

Chen

et al. 2024

Hum Genomics

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Objective Myasthenia gravis (MG) is a complex autoimmune disease affecting the neuromuscular junction with limited drug options, but the field of MG treatment recently benefits from novel biological agents. We performed a drug-targeted Mendelian randomization (MR) study to identify novel therapeutic targets of MG. Methods Cis-expression quantitative loci (cis-eQTL), which proxy expression levels for 2176 druggable genes, were used for MR analysis. Causal relationships between genes and disease, identified by eQTL MR analysis, were verified by comprehensive sensitivity, colocalization, and protein quantitative loci (pQTL) MR analyses. The protein-protein interaction (PPI) analysis was also performed to extend targets, followed by enzyme-linked immunosorbent assay (ELISA) to explore the serum level of drug targets in MG patients. A phenome-wide MR analysis was then performed to assess side effects with a clinical trial review assessing druggability. Results The eQTL MR analysis has identified eight potential targets for MG, one for early-onset MG and seven for late-onset MG. Further colocalization analyses indicated that CD226, CDC42BPB, PRSS36, and TNFSF12 possess evidence for colocalization with MG or late-onset MG. pQTL MR analyses identified the causal relations of TNFSF12 and CD226 with MG and late-onset MG. Furthermore, PPI analysis has revealed the protein interaction between TNFSF12-TNFSF13(APRIL) and TNFSF12-TNFSF13B(BLyS). Elevated TNFSF13 serum level of MG patients was also identified by ELISA experiments. This study has ultimately proposed three promising therapeutic targets (TNFSF12, TNFSF13, TNFSF13B) of MG. Conclusions Three drug targets associated with the BLyS/APRIL pathway have been identified. Multiple biological agents, including telitacicept and belimumab, are promising for MG therapy.

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Section: Discussionmentioning

confidence: 99%