About: Modulation of metabolic functions through Cas13d-mediated gene knockdown in liver

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An Entity of Type : schema:ScholarlyArticle, within Data Space : covidontheweb.inria.fr associated with source document(s)

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type	Academic Article research paper schema:ScholarlyArticle
isDefinedBy	Covid-on-the-Web dataset
title	Modulation of metabolic functions through Cas13d-mediated gene knockdown in liver
Creator	Liu, Jing Yang, Hui Li, Zhijie Huang, Jia He, Bingbing »more»
source	BioRxiv; MedRxiv
abstract	RNA knockdown in vivo carries significant potential for disease modelings and therapies. Despite the emerging approaches of CRISPR/Cas9-mediated permanent knock out of targeted genes, strategies targeting RNA for disruption are advantageous in the treatment of acquired metabolic disorders when permanent modification of the genome DNA is not appropriate, and RNA virus infection diseases when pathogenic DNA is not available (such as SARS-Cov-2 and MERS infections). Recently, Cas13d, a family of RNA-targeting CRISPR effectors, has been shown to accomplish robust down-regulation of cellular RNAs in mammalian cells in vitro. Among the various Cas13d subtypes, CasRx (RfxCas13d) showed the most potent RNA knockdown efficiency in HEK293T cells. However, the RNA-targeting activity of Cas13d still needs to be verified in vivo. In this study, the CasRx system was demonstrated to efficiently and functionally knock down genes related to metabolism functions, including Pten, Pcsk9 and lncLstr, in mouse hepatocytes. CasRx-mediated simultaneous knockdown of multiple genes was also achieved by sgRNA arrays, providing a useful strategy to modulate complex metabolism networks. Moreover, the AAV (adeno-associated virus)-mediated delivery of CasRx and Pcsk9 sgRNAs into mouse liver successfully decreased serum PCSK9, resulting in significant reduction of serum cholesterol levels. Importantly, CasRx-mediated knockdown of Pcsk9 is reversible and Pcsk9 could be repeatedly down-regulated, providing an effective strategy to reversibly modulate metabolic genes. The present work supplies a successful proof-of-concept trial that suggests efficient and regulatory knockdown of target metabolic genes for a designed metabolism modulation in the liver.
has issue date	2020-02-19(xsd:dateTime)
bibo:doi	10.1101/2020.02.17.945014
has license	biorxiv
sha1sum (hex)	0eed0db625a4520c7ae52aefe10bae3417d31c7f
schema:url	https://doi.org/10.1101/2020.02.17.945014
resource representing a document's title	Modulation of metabolic functions through Cas13d-mediated gene knockdown in liver
schema:publication	bioRxiv
resource representing a document's body	covid:0eed0db625a4520c7ae52aefe10bae3417d31c7f#body_text
is schema:about of	named entity 'permanent' named entity 'PCSK9' named entity 'diseases' named entity 'knockdown' named entity 'Pcsk9' »more»

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