Using human pluripotent stem cell models to study autism in the era of big data.
Mol Autism
; 11(1): 21, 2020 03 23.
Article
em En
| MEDLINE
| ID: mdl-32293529
ABSTRACT
Advances in human pluripotent stem cell (hPSC) biology coupled with protocols to generate diverse brain cell types in vitro have provided neuroscientists with opportunities to dissect basic and disease mechanisms in increasingly relevant cellular substrates. At the same time, large data collections and analyses have facilitated unprecedented insights into autism genetics, normal human genetic variation, and the molecular landscape of the developing human brain. While such insights have enabled the investigation of key mechanistic questions in autism, they also highlight important limitations associated with the use of existing hPSC models. In this review, we discuss four such issues which influence the efficacy of hPSC models for studying autism, including (i) sources of variance, (ii) scale and format of study design, (iii) divergence from the human brain in vivo, and (iv) regulatory policies and compliance governing the use of hPSCs. Moreover, we advocate for a set of immediate and long-term priorities to address these issues and to accelerate the generation and reproducibility of data in order to facilitate future fundamental as well as therapeutic discoveries.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transtorno Autístico
/
Células-Tronco Pluripotentes
/
Modelos Biológicos
Tipo de estudo:
Guideline
Limite:
Animals
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Humans
Idioma:
En
Revista:
Mol Autism
Ano de publicação:
2020
Tipo de documento:
Article
País de afiliação:
Estados Unidos