Small Molecule Therapy for Genetic Disease


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The genetic determinants of many diseases, including monogenic diseases and cancers, have been identified; nevertheless, targeted therapy remains elusive for most. High-throughput screening HTS of small molecules, including high-content analysis HCA , has been an important technology for the discovery of molecular tools and new therapeutics. HTS can be based on modulation of a known disease target called reverse chemical genetics or modulation of a disease-associated mechanism or phenotype forward chemical genetics.


  1. Organization of the Grand Lodge of England.
  2. VERORDNUNG (EG) Nr. 440/2008 DER KOMMISSION: REACH Testmethoden (German Edition)?
  3. Proceedings of the American Thoracic Society;

Prominent target-based successes include modulators of transthyretin, used to treat transthyretin amyloidoses, and the BCR-ABL kinase inhibitor Gleevec, used to treat chronic myelogenous leukemia. Phenotypic screening successes include modulators of cystic fibrosis transmembrane conductance regulator, splicing correctors for spinal muscular atrophy, and histone deacetylase inhibitors for cancer. Synthetic lethal screening, in which chemotherapeutics are screened for efficacy against specific genetic backgrounds, is a promising approach that merges phenotype and target.

In this article, we introduce HTS technology and highlight its contributions to the discovery of drugs and probes for monogenic diseases and cancer. HCA; HTS; cancer; drug discovery; high-content analysis; high-throughput screening; monogenic disease; small molecules. Small Molecular Therapy for Genetic Diseases is a collection of articles that commemorate the Orphan Drug Act that was passed in after sustained effort from patient advocacy groups.

Small Molecules to Treat Cystic Fibrosis | Proceedings of the American Thoracic Society

The legislation gave pharmaceutical companies various incentives, such as tax credits, expedited review, flexible clinical trial requirements, and exclusive marketing to develop treatments that would otherwise be unprofitable. Another significant portion of drugs are cytotoxic agents for malignancies or autoimmune diseases. Only 24 agents are small molecule drugs for genetic diseases, but it is this category of drugs that provides the focus for the rest of the book.

Although macromolecular therapy may dominate the post-genomic world, the authors remind us that small molecules have many distinct advantages, such as easy delivery, straightforward pharmacokenitics, and minimal immune interactions.

After an introductory section covering legislative backgrounds, infrastructures, and pharmacology principles, the book reviews 11 examples of effective small molecule therapy. In each chapter, an overview of the pathogenesis and epidemiology of the condition is followed by clinical trial and case study data of the drug. The book targets three types of readers: clinicians and clinical scientists, patients and patient advocates, and policy makers.

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The sense of hope and encouragement that permeates the pages enables the book to motivate physicians and researchers to use and develop small molecule therapies. But someone familiar with biomedical sciences will likely find the articles rather rudimentary.

Small-Molecule Probes: Bridging the gap between understanding and curing disease

Its important introductory chapters discuss the infrastructure of the field. The book closely analyses the cofactors used to augment the function of defective enzymes and the compounds that are able to utilise an alternative pathway in order to avoid the consequences of the metabolic block present in the patient. Among other therapies, the authors discuss the use of zinc and tetrathiomolybdate to treat Wilson's disease and the use of cysteamine to treat nephropathic cystinosis.


  • Synthetic small-molecule RNA ligands: future prospects as therapeutic agents.
  • Publication details.
  • Small-Molecule Screening for Genetic Diseases. - PubMed - NCBI!
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    Small Molecule Therapy for Genetic Disease Small Molecule Therapy for Genetic Disease
    Small Molecule Therapy for Genetic Disease Small Molecule Therapy for Genetic Disease
    Small Molecule Therapy for Genetic Disease Small Molecule Therapy for Genetic Disease
    Small Molecule Therapy for Genetic Disease Small Molecule Therapy for Genetic Disease
    Small Molecule Therapy for Genetic Disease Small Molecule Therapy for Genetic Disease
    Small Molecule Therapy for Genetic Disease Small Molecule Therapy for Genetic Disease
    Small Molecule Therapy for Genetic Disease Small Molecule Therapy for Genetic Disease

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