Target intelligence / Profile preview

Muscleblind-like protein 1 (MBNL1)

Target: MBNL1
Molecular classification: RNA-binding protein, Transcription factor

Overview

Muscleblind-like protein 1 (MBNL1) is an RNA-binding protein crucial for alternative splicing regulation, mRNA processing, and microRNA biogenesis. In myotonic dystrophy (DM1/DM2), MBNL1 is abnormally sequestered within nuclear foci due to binding with expanded CUG or CCUG repeat-containing RNAs, leading to loss-of-function and disease pathology.

Mechanism of action

Disrupting toxic RNA-protein interactions (therapeutic strategies)

Biological functions

Alternative splicing regulationmRNA processingMicroRNA biogenesis

Disease associations

Myotonic dystrophy type 1Myotonic dystrophy type 2

Safety considerations

Releasing sequestered protein without proper control could lead to unintended splicing changes.

Biomarkers

Nuclear foci of MBNL1 (in DM1/DM2)Mis-splicing events (e.g., chloride channel, troponin T isoforms)

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