PXD012581

PXD012581 is an original dataset announced via ProteomeXchange.

Title	Engineered transfer RNAs for suppression of premature termination codons
Description	PTCs cause a multitude of human diseases and there are no established therapeutic options for their therapeutic management. Herein, we report the high-throughput cloning and identification, characterization and functional analysis of anticodon-edited tRNA which display efficacious PTC reversion in eukaryotic cells and mouse skeletal muscle. Notably, our screen identifies ACE-tRNA, in total, with the potential to repair a vast majority of known human disease-causing PTC, but this therapeutic will require overcoming tissue and delivery specific challenges. However, the engineered tRNA, once delivered, faithfully encode their cognate amino acid, thus abrogating spurious effects on downstream protein stability, folding, and trafficking, and consequently negating the need for tandem therapies involving protein folding or trafficking agents. When transfected as cDNA, ACE-tRNAs rescued multiple full-length proteins via PTC suppression; a NLuc luciferase reporter, a model protein HDH, and two disease nonsense mutations in CFTR.
HostingRepository	PRIDE
AnnounceDate	2019-02-13
AnnouncementXML	Submission_2019-02-13_04:38:27.xml
DigitalObjectIdentifier
ReviewLevel	Peer-reviewed dataset
DatasetOrigin	Original dataset
RepositorySupport	Unsupported dataset by repository
PrimarySubmitter	robert Pope
SpeciesList	scientific name: Escherichia coli; NCBI TaxID: 562; scientific name: Homo sapiens (Human); NCBI TaxID: 9606;
ModificationList	monohydroxylated residue; iodoacetamide derivatized residue
Instrument	Orbitrap Fusion Lumos

Revision	Datetime	Status	ChangeLog Entry
0	2019-02-01 01:11:30	ID requested
⏵ 1	2019-02-13 04:38:28	announced

Dataset with its publication pending

curator keyword: Biological

submitter keyword: PTC repair, anticodon edited tRNAs, ACE-tRNAs

Robert Marshall Pope
contact affiliation	[1]University of Iowa, Carver College of Medicine, Iowa City, Iowa, 52242, USA [2]University of Rochester, Rochester, NY, 14642
contact email	marshall-pope@uiowa.edu
lab head
robert Pope
contact affiliation	University of Iowa, COM, Iowa City, IA 52241
contact email	marshall-pope@uiowa.edu
dataset submitter

Dataset FTP location NOTE: Most web browsers have now discontinued native support for FTP access within the browser window. But you can usually install another FTP app (we recommend FileZilla) and configure your browser to launch the external application when you click on this FTP link. Or otherwise, launch an app that supports FTP (like FileZilla) and use this address: ftp://ftp.pride.ebi.ac.uk/pride/data/archive/2019/02/PXD012581

PRIDE project URI

• PRIDE
  1. PXD012581
     1. Label: PRIDE project
     2. Name: Engineered transfer RNAs for suppression of premature termination codons