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A human-on-a-chip approach to tackling rare diseases

de Mello, Camilly P. Pires ; Rumsey, John ; Slaughter, Victoria ; Hickman, James J.

Drug discovery today, 2019-11, Vol.24 (11), p.2139-2151 [Periódico revisado por pares]

England: Elsevier Ltd

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  • Título:
    A human-on-a-chip approach to tackling rare diseases
  • Autor: de Mello, Camilly P. Pires ; Rumsey, John ; Slaughter, Victoria ; Hickman, James J.
  • É parte de: Drug discovery today, 2019-11, Vol.24 (11), p.2139-2151
  • Notas: ObjectType-Article-1
    SourceType-Scholarly Journals-1
    ObjectType-Feature-3
    content type line 23
    ObjectType-Review-2
    Authors contributed equally to this work.
  • Descrição: •Drug development and disease modeling for rare diseases remains a challenge.•Advances in in vitro modeling has led to development of human-on-a-chip technology.•PK and PD mathematical modeling is a powerful tool for orphan drug development.•HoaC systems have potential for facilitating rare disease and orphan drug research. Drug development for rare diseases, classified as diseases with a prevalence of < 200 000 patients, is limited by the high cost of research and low target population. Owing to a lack of representative disease models, research has been challenging for orphan drugs. Human-on-a-chip (HoaC) technology, which models human tissues in interconnected in vitro microfluidic devices, has the potential to lower the cost of preclinical studies and increase the rate of drug approval by introducing human phenotypic models early in the drug discovery process. Advances in HoaC technology can drive a new approach to rare disease research and orphan drug development. After more than three decades of the Orphan Drug Act, drug development for rare diseases remains a challenge. Human-on-a-chip technology holds the promise to drive orphan drug development to the next level.
  • Editor: England: Elsevier Ltd
  • Idioma: Inglês
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