CRISPR Gene-Editing Models Geared Toward Therapy for Hereditary and Developmental Neurological Disorders.
Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)
caffeine
drug responsiveness
gene therapy
gene-editing
hereditary neurological disorders
neonates
pharmacogenomics
Journal
Frontiers in pediatrics
ISSN: 2296-2360
Titre abrégé: Front Pediatr
Pays: Switzerland
ID NLM: 101615492
Informations de publication
Date de publication:
2021
2021
Historique:
received:
07
08
2020
accepted:
19
02
2021
entrez:
1
4
2021
pubmed:
2
4
2021
medline:
2
4
2021
Statut:
epublish
Résumé
Hereditary or developmental neurological disorders (HNDs or DNDs) affect the quality of life and contribute to the high mortality rates among neonates. Most HNDs are incurable, and the search for new and effective treatments is hampered by challenges peculiar to the human brain, which is guarded by the near-impervious blood-brain barrier. Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR), a gene-editing tool repurposed from bacterial defense systems against viruses, has been touted by some as a panacea for genetic diseases. CRISPR has expedited the research into HNDs, enabling the generation of
Identifiants
pubmed: 33791256
doi: 10.3389/fped.2021.592571
pmc: PMC8006930
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
592571Informations de copyright
Copyright © 2021 Wong, Cheah, Syafruddin, Mohtar, Azmi, Ng and Chua.
Déclaration de conflit d'intérêts
The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
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