Key result
Misfolded sarcoglycans responsible for sarcoglycanopathies could potentially be rescued to the cell membrane by pharmacological agents that assist their maturation along the ER secretory pathway.
Population
Patients with sarcoglycanopathies and related animal/cell models
Design
Review
Authors
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Hypothesis-generating for ER-targeted rescue in sarcoglycanopathies; prospective trials required before clinical consideration.
The ER quality control machinery represents a potential pharmacological target for rescuing misfolded proteins in sarcoglycanopathies.
Sandonà et al. (2009) conducted a review in Sarcoglycanopathies (Limb-girdle muscular dystrophies 2C-2F). Pharmacological interference with ER quality control machinery was evaluated. Misfolded sarcoglycans responsible for sarcoglycanopathies could potentially be rescued to the cell membrane by pharmacological agents that assist their maturation along the ER secretory pathway.
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