Protein Homeostasis Diseases: Mechanisms and Novel Therapies offers
an interdisciplinary examination of the fundamental aspects,
biochemistry and molecular biology of protein homeostasis disease,
including the use of natural and pharmacological small molecules to
treat common and rare protein homeostasis disorders. Contributions
from international experts discuss the biochemical and genetic
components of protein homeostasis disorders, the mechanisms by
which genetic variants may cause loss-of-function and
gain-of-toxic-function, and how natural ligands can restore protein
function and homeostasis in genetic diseases. Applied chapters
provide guidance on employing high throughput sequencing and
screening methodologies to develop pharmacological chaperones and
repurpose approved drugs to treat protein homeostasis disorders.
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