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Targeting Wolman Disease and Cholesteryl Ester Storage Disease: Disease Pathogenesis and Therapeutic Development

Francis Aguisanda1, Natasha Thorne1, Wei Zheng1

  • 1National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, MD 20892-3370, USA.

Current Chemical Genomics and Translational Medicine
|April 13, 2017
PubMed

Keywords:
Cell-based disease modelCholesteryl ester storage diseaseHigh-throughput screeningInduced pluripotent stem cellsLysosomal storage diseaseWolman disease

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