Chemogenetics: drug-controlled gene therapies for neural circuit disorders.
Journal
Cell & gene therapy insights
ISSN: 2059-7800
Titre abrégé: Cell Gene Ther Insights
Pays: England
ID NLM: 101712177
Informations de publication
Date de publication:
Aug 2020
Aug 2020
Historique:
entrez:
23
8
2021
pubmed:
24
8
2021
medline:
24
8
2021
Statut:
ppublish
Résumé
Many patients with nervous system disorders have considerable unmet clinical needs or suffer debilitating drug side effects. A major limitation of exiting treatment approaches is that traditional small molecule pharmacotherapy lacks sufficient specificity to effectively treat many neurological diseases. Chemogenetics is a new gene therapy technology that targets an engineered receptor to cell types involved in nervous system dysfunction, enabling highly selective drug-controlled neuromodulation. Here, we discuss chemogenetic platforms and considerations for their potential application as human nervous system therapies.
Identifiants
pubmed: 34422319
doi: 10.18609/cgti.2020.112
pmc: PMC8376173
mid: NIHMS1707669
doi:
Types de publication
Journal Article
Langues
eng
Pagination
1079-1094Subventions
Organisme : Howard Hughes Medical Institute
Pays : United States
Déclaration de conflit d'intérêts
Disclosure and potential conflicts of interest: Scott M Sternson receives research funding from the Howard Hughes Medical Institute. He is a co-founder and paid consultant for Redpin Therapeutics, which is developing chemogenetic therapies for nervous system disorders. He has issued and pending patents for PSAM-based chemogenetic ion channels. David Bleakman is CSO of Redpin Therapeutics.
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