Article
Role of HSP70 chaperone in protein aggregate phenomenon of GNE mutant cells: Therapeutic lead for GNE Myopathy.
The international journal of biochemistry & cell biology - 1 Aug 2022
Yadav Rashmi, Devi Shamulailatpam Shreedarshanee, Oswalia Jyoti, Ramalingam Sivaprakash, Arya Ranjana
Abstract excerpt
Limited treatment options and research in understanding the pathomechanisms of rare diseases has raised concerns about their therapeutic development. One such poorly understood ultra-rare neuromuscular disorder is GNE Myopathy (GNEM) which is caused due to mutation in key sialic acid biosynthetic enzyme, GNE. Treatment with sialic acid or its derivatives/precursors slows the disease progression, but curative...
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