Human induced pluripotent stem cells (hiPSCs) were first generated in 2007, but the full translational potential of this valuable tool has yet to be realized. The potential applications of hiPSCs are especially relevant to neurology, as brain cells from patients are rarely available for research. hiPSCs from individuals with neuropsychiatric or neurodegenerative diseases have facilitated biological and multi-omics studies as well as large-scale screening of chemical libraries. However, researchers are struggling to improve the scalability, reproducibility and quality of this descriptive disease modelling. Addressing these limitations will be the first step towards a new era in hiPSC research — that of predictive disease modelling — involving the correlation and integration of in vitro experimental data with longitudinal clinical data. This approach is a key element of the emerging precision medicine paradigm, in which hiPSCs could become a powerful diagnostic and prognostic tool. Here, we consider the steps necessary to achieve predictive modelling of neurodegenerative disease with hiPSCs, using Huntington disease as an example.

hiPSCs for predictive modelling of neurodegenerative diseases : dreaming the possible / P. Rivetti di Val Cervo, D. Besusso, P. Conforti, E. Cattaneo. - In: NATURE REVIEWS. NEUROLOGY. - ISSN 1759-4758. - 17:6(2021), pp. 381-392. [10.1038/s41582-021-00465-0]

hiPSCs for predictive modelling of neurodegenerative diseases : dreaming the possible

P. Rivetti di Val Cervo
;
D. Besusso;P. Conforti;E. Cattaneo
2021

Abstract

Human induced pluripotent stem cells (hiPSCs) were first generated in 2007, but the full translational potential of this valuable tool has yet to be realized. The potential applications of hiPSCs are especially relevant to neurology, as brain cells from patients are rarely available for research. hiPSCs from individuals with neuropsychiatric or neurodegenerative diseases have facilitated biological and multi-omics studies as well as large-scale screening of chemical libraries. However, researchers are struggling to improve the scalability, reproducibility and quality of this descriptive disease modelling. Addressing these limitations will be the first step towards a new era in hiPSC research — that of predictive disease modelling — involving the correlation and integration of in vitro experimental data with longitudinal clinical data. This approach is a key element of the emerging precision medicine paradigm, in which hiPSCs could become a powerful diagnostic and prognostic tool. Here, we consider the steps necessary to achieve predictive modelling of neurodegenerative disease with hiPSCs, using Huntington disease as an example.
Settore BIO/14 - Farmacologia
H20_RIA20ECATT_01 - Novel Strategies for Cell-based Neural Reconstruction (NSC-Reconstruct) - CATTANEO, ELENA - H20_RIA - Horizon 2020_Research & Innovation Action/Innovation Action - 2020
Article (author)
File in questo prodotto:
File Dimensione Formato  
s41582-021-00465-0.pdf

accesso aperto

Tipologia: Publisher's version/PDF
Dimensione 2.3 MB
Formato Adobe PDF
2.3 MB Adobe PDF Visualizza/Apri
Pubblicazioni consigliate

Caricamento pubblicazioni consigliate

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/877961
Citazioni
  • ???jsp.display-item.citation.pmc??? 7
  • Scopus 9
  • ???jsp.display-item.citation.isi??? 8
social impact