Journal
ACS CHEMICAL BIOLOGY
Volume 11, Issue 6, Pages 1471-1489Publisher
AMER CHEMICAL SOC
DOI: 10.1021/acschembio.6b00195
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Funding
- National Institutues of Health [R01GM080742]
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Errors in protein, folding, may result in premature clearance of structurally aberrant proteins, or in the, accumulation of toxic misfolded species or protein aggregates. These pathological events lead to a large range of conditions known as Conformational diseases. Several research groups have presented possible therapeutic solutions, for their treatment by developing,novel compounds, known as: pharmacological chaperones.. These cell-permeable molecules selectively provide a molecular scaffold around which misfolded proteins can recover their native folding and; thus, their 'biological activities. Here, we review therapeutic Strategies, clinical potentials, and cost-benefit impacts of several classes of pharmacological chaperones,for the:treatment of a, series of conformational diseases.
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