Pharmacological Chaperones: Design and Development of New Therapeutic Strategies for the Treatment of Conformational Diseases

Marino Convertino, Jhuma Das, Nikolay V. Dokholyan

Research output: Contribution to journalReview articlepeer-review

51 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)1471-1489
Number of pages19
JournalACS chemical biology
Volume11
Issue number6
DOIs
StatePublished - Jun 17 2016

All Science Journal Classification (ASJC) codes

  • Biochemistry
  • Molecular Medicine

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