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Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
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Related Experiment Video

Updated: Feb 23, 2026

Ion Mobility-Mass Spectrometry Techniques for Determining the Structure and Mechanisms of Metal Ion Recognition and Redox Activity of Metal Binding Oligopeptides
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Wilson's disease: Prospective developments towards new therapies.

Giusy Ranucci1, Roman Polishchuck1, Raffaele Iorio1

  • 1Giusy Ranucci, Raffaele Iorio, Department of Translational Medical Science, Section of Pediatric, University Federico II, 80131 Naples, Italy.

World Journal of Gastroenterology
|August 31, 2017
PubMed
Summary

New Wilson's disease (WD) therapies are being developed to address limitations of current treatments. Research focuses on correcting ATP7B gene mutations and finding new compounds for a safer, more effective cure.

Keywords:
ATP7BCorrectorsHeat shock protein 70JNKMethanobactinPrecision medicineStem cell-derived hepatocyte like cellsTranslational medicinep38

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Area of Science:

  • Genetics and Metabolism
  • Molecular Biology

Background:

  • Wilson's disease (WD) is an inherited disorder of copper metabolism caused by mutations in the ATP7B gene.
  • Current treatments like zinc salts and copper chelators are effective but have limitations due to side effects and patient intolerance.

Discussion:

  • Novel therapeutic strategies are emerging, including gene correction, new compound discovery, and cell/gene therapies.
  • These approaches aim to circumvent ATP7B deficiency and offer improved treatment options for WD patients.

Key Insights:

  • Research is exploring ways to correct ATP7B gene mutations directly.
  • Discovery of new compounds that bypass the need for a functional ATP7B protein is a key focus.
  • Cell and gene therapies represent promising avenues for treating Wilson's disease.

Outlook:

  • Translational research is crucial to bring these novel therapies into clinical practice.
  • Meeting clinical trial requirements is essential for the successful development of next-generation WD treatments.
  • Advancements in understanding WD pathogenesis provide a foundation for developing tolerable and safe cures.