Browse
Submit Data
Databases
API
Help

Dataset Information

0 Views

0 Connections

0 Citations

0 Reanalyses

0 Downloads

Omics score: 0

Heart-specific gene silencing via single-AAV-delivered base editing [Amplicon-Seq]

ABSTRACT: Heart-specific gene silencing via single-AAV-delivered base editing [Amplicon-Seq]

PROVIDER: PRJNA1108200 | ENA |

REPOSITORIES: ENA

ACCESS DATA

Json Xml

Dataset's files

Source:

			Action	DRS
	SRR28906600_1.fastq.gz	Fastqsanger.gz
	SRR28906600_2.fastq.gz	Fastqsanger.gz
	SRR28906601_1.fastq.gz	Fastqsanger.gz
	SRR28906601_2.fastq.gz	Fastqsanger.gz
	SRR28906602_1.fastq.gz	Fastqsanger.gz

Items per page:

1 - 5 of 508

Similar Datasets

Heart-specific gene silencing via single-AAV-delivered base editing [RNA-Seq]

Project description:Heart-specific gene silencing via single-AAV-delivered base editing [RNA-Seq]

| PRJNA1104998 | ENA

Heart-specific gene silencing via single-AAV-delivered base editing [Amplicon-Seq]

Project description:We used an adenine base editor to target the translation start site and mRNA splicing site of Camk2d in order to knock out CaMKIIδ. We found that editing the 5' splice site of intron 7 can lead to premature translation termination, effectively knocking out CaMKIIδ.

2025-01-21 | GSE266652 | GEO

Heart-specific gene silencing via single-AAV-delivered base editing [RNA-Seq]

2025-01-21 | GSE265935 | GEO

AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (amplicon)

Project description:AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (amplicon)

| PRJNA743041 | ENA

AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (amplicon)

Project description:In this study, we utilized a mouse model of mucopolysaccharidosis type I (MPS-I) carrying a homozygous nonsense mutation in the Idua gene (Idua-W401X, TGG→TAG) to develop rAAV-delivered sup-tRNA therapeutics that can overcome a pathogenic UAG premature termination codon (PTC).

2021-11-30 | GSE179270 | GEO

RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes

Project description:Adeno-associated virus (AAV)-based gene therapy could be facilitated by the development of molecular switches to control the magnitude and timing of expression of therapeutic transgenes. RNA interference (RNAi)-based approaches hold unique potential as a clinically proven modality to pharmacologically regulate AAV gene dosage in a sequence-specific manner. We present a generalizable RNAi-based rheostat wherein AAV transgene expression is silenced using the clinically validated modality of chemically modified short interfering RNA (siRNA) conjugates or vectorized co-expression of short hairpin RNA (shRNA). For transgene induction, we employ REVERSIR technology, a synthetic high-affinity oligonucleotide complementary to the siRNA or shRNA guide strand to reverse RNAi activity and rapidly recover transgene expression. For potential clinical development, we report potent and specific siRNA sequences that may allow selective regulation of transgenes while minimizing unintended off-target effects. Our results establish a conceptual framework for RNAi-based regulatory switches with potential for infrequent dosing in clinical settings to dynamically modulate expression of virally-delivered gene therapies.

2023-01-31 | GSE214065 | GEO

AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice

Project description:AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice

| PRJNA743034 | ENA

RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes

Project description:RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes

| PRJNA883694 | ENA

Mitochondrial DNA base editing in vivo via viral DdCBE delivery to somatic cells

Project description:We show that delivering the mitochondrial base editor DdCBEs via AAV transduction of somatic cells efficiently produces precise base editing of the intended region.

2022-01-19 | GSE184064 | GEO

AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (tRNA_charging)

Project description:AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice (tRNA_charging)

| PRJNA781652 | ENA