Target intelligence / Profile preview

Muscle-specific kinase B-cell receptor and DNA (MuSK BCR/DNA)

Target
MuSK BCR/DNA
Molecular classification
B-cell receptor, Nucleic acid, Receptor
01

Overview

This target entry is a composite description typically associated with therapeutic strategies for Muscle-Specific Kinase (MuSK) Myasthenia Gravis, an autoimmune disorder where patients produce antibodies against MuSK at the neuromuscular junction (PMID: 31243143). The first component, the Anti-MuSK B-cell receptor, is a highly specific target for Chimeric Autoantibody Receptor (CAAR) T-cell therapies, such as MuSK-CAART, which are designed to selectively bind and kill only the B-cells expressing receptors for MuSK while sparing healthy B-cells (PMID: 31243143, ClinicalTrials.gov: NCT04422912). The second component, broad DNA in proliferating lymphocytes, refers to the mechanism of traditional immunosuppressive agents like cyclophosphamide or mycophenolate, which disrupt DNA replication to prevent the expansion of all activated lymphocytes. This combination entry likely reflects a clinical protocol or a multi-pronged pharmacological approach aimed at both the precision depletion of autoantibody-producing cells and the broad suppression of the inflammatory immune environment.

Other names
Anti-MuSK B-cell receptorMuSK-specific B-cell receptorDNA in proliferating lymphocytesMuscle-specific tyrosine kinase receptor (MuSK) B-cell receptor
02

Mechanism of action

The target represents a dual approach: specific elimination of pathogenic B-cells via Chimeric Autoantibody Receptor (CAAR) T-cells that recognize the anti-MuSK B-cell receptor, and non-specific suppression of lymphocyte proliferation through the inhibition of DNA synthesis or induction of DNA damage.

03

Biological functions

Immune responseB-cell activationCell proliferationDNA replication
04

Disease associations

Myasthenia gravisAutoimmune diseaseNeuromuscular junction disorders
05

Safety considerations

Cytokine release syndrome (CRS)NeurotoxicityB-cell aplasiaBone marrow suppressionIncreased risk of infection
06

Interacting drugs

MuSK-CAART (DSG-101)

2 more in the full profile.

07

Biomarkers

Anti-MuSK autoantibodiesMuSK-specific B-cell frequencyLymphocyte count

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