Browse
Submit Data
Databases
API
Help

Dataset Information

0 Views

0 Connections

0 Citations

0 Reanalyses

0 Downloads

Omics score: 0

ABSTRACT: In vivo adenine base editing in an adult mouse model of tyrosinemia

PROVIDER: PRJNA513076 | ENA |

REPOSITORIES: ENA

ACCESS DATA

Json Xml

Dataset's files

Source:

			Action	DRS
	SRR8397420.fastq.gz	Fastqsanger.gz
	SRR8397417_1.fastq.gz	Fastqsanger.gz
	SRR8397417_2.fastq.gz	Fastqsanger.gz
	SRR8397418.fastq.gz	Fastqsanger.gz
	SRR8397419.fastq.gz	Fastqsanger.gz

Items per page:

1 - 5 of 5

Similar Datasets

Humans mice

Project description:Adenine base editing in an adult mouse model of tyrosinemia

| PRJNA513291 | ENA

Project description:In vivo Adenine Base Editing rescues adrenoleukodystrophy in a humanized mouse model

| PRJNA1020620 | ENA

Project description:Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

| PRJNA451048 | ENA

In vivo adenine base editing of PCSK9 in mice and macaques reduces LDL-cholesterol levels [amplicon-seq]

Project description:The majority of known pathogenic point mutations in the human genome are C•G to T•A substitutions. Adenine base editors (ABEs), comprised of nuclease-impaired Cas9 fused to adenine deaminases, enable direct repair of these mutations, making them promising tools for precision in vivo genome editing therapies. However, prior to application in patients, thorough safety and efficacy studies in relevant model organisms are needed. Here, we apply adenine base editing in vivo in the liver of mice and cynomolgus macaques to install a splice site mutation in PCSK9 and reduce blood low-density lipoprotein (LDL) levels, a well-known risk factor for cardiovascular disease. Intravenous delivery of ABE-encoding mRNA and a locus-specific single guide (sg)RNA utilizing lipid nanoparticle (LNP) technology induce up to 67% editing in the liver of mice and up to 34% editing in the liver of macaques, leading to a reduction of plasma PCSK9 and LDL levels. We observed rapid clearance of ABE mRNA after LNP-mediated delivery, and neither sgRNA-dependent nor sgRNA-independent off-target mutations are detected in genomic DNA. Together, our findings support safety and feasibility of adenine base editing to treat patients with monogenetic liver diseases.

2021-04-13 | GSE168364 | GEO

In vivo adenine base editing of PCSK9 in mice and macaques reduces LDL-cholesterol levels [RNA-seq]

2021-04-13 | GSE168363 | GEO

Project description:Adenine Base Editing in iPSCs

| PRJNA970697 | ENA

Mus musculus

Project description:Ex vivo therapeutic base and prime editing using chemically derived hepatic progenitors in a mouse model of tyrosinemia type 1

| PRJNA717639 | ENA

Project description:In Vivo Adenine Base Editing Corrects Hutchinson - Gilford Progeria Syndrome

| PRJNA627465 | ENA

Project description:A split and inducible adenine base editor for precise in vivo base editing

| PRJNA923001 | ENA

Bimodal Adenine and Cytosine CRISPR Base Editing on DNA

Project description:Adenine and cytosine base editors (ABEs and CBEs) represent a new genome editing technology that allows the programmable installation of A-to-G or C-to-T alterations on DNA. We engineered Streptococcus pyogenes Cas9-based adenine and cytosine base editor (SpACE) that enables efficient simultaneous introduction of A-to-G and C-to-T substitutions in the same base editing window on DNA.

2020-07-02 | GSE137411 | GEO

OmicsDI is part of the ELIXIR infrastructure

OmicsDI is an Elixir interoperability service. Learn more ›

Tweets

OmicsDI Databases

PRIDE
PeptideAtlas
MassIVE
JPOST Repository
Physiome Model Repository

EGA
EVA
ENA
LINCS
PAXDB
Cell Collective

MetaboLights
Metabolomics Workbench
MetabolomeExpress
GNPS
BioModels
FAIRDOMHub

ArrayExpress
dbGaP
ExpressionAtlas
GEO
NODE

Information

Databases
Help
API
Contact us
Code on GitHub
Terms of use
Submit Data