Browse
Submit Data
Databases
API
Help

Dataset Information

0 Views

0 Connections

0 Citations

0 Reanalyses

0 Downloads

Omics score: 0

In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model

ABSTRACT: In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model

PROVIDER: PRJNA879222 | ENA |

REPOSITORIES: ENA

ACCESS DATA

Json Xml

Dataset's files

Source:

			Action	DRS
	SRR21526500_1.fastq.gz	Fastqsanger.gz
	SRR21526500_2.fastq.gz	Fastqsanger.gz
	SRR21526501_1.fastq.gz	Fastqsanger.gz
	SRR21526501_2.fastq.gz	Fastqsanger.gz
	SRR21526502_1.fastq.gz	Fastqsanger.gz

Items per page:

1 - 5 of 160

Similar Datasets

In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model

Project description:CRISPR-based gene editing technology represents a promising approach to deliver therapies for inherited disorders, including amyotrophic lateral sclerosis (ALS). Toxic gain-of-function superoxide dismutase 1 (SOD1) mutations are responsible for ~20% of familial ALS cases. Thus, current clinical strategies to treat SOD1-ALS are designed to lower SOD1 levels. Here, we utilized AAV-PHP.B variants to deliver CRISPR-Cas9 guide RNAs designed to disrupt the human SOD1 (huSOD1) transgene in SOD1G93A mice. A one-time intracerebroventricular injection of AAV.PHP.B-huSOD1-sgRNA into neonatal H11Cas9 SOD1G93A mice caused robust and sustained mutant huSOD1 protein reduction in the cortex and spinal cord, and restored motor function. Neonatal treatment also reduced spinal motor neuron loss, neuromuscular junction (NMJ) denervation and muscle atrophy, diminished axonal damage and preserved compound muscle action potential throughout the lifespan of treated mice. SOD1G93A treated mice achieved significant disease-free survival, extending lifespan by more than 110 days. Importantly, a one-time intrathecal or intravenous injection of AAV.PHP.eB-huSOD1-sgRNA in adult H11Cas9 SOD1G93A mice, immediately before symptom onset, also extended lifespan by at least 170 days. We observed substantial protection against disease progression demonstrating the utility of our CRISPR editing preclinical approach for target evaluation. Our work not only uncovered key parameters (e.g., AAV capsid, Cas9 expression) that resulted in improved efficacy compared similar approaches but can also serve to accelerate drug target validation.

2022-12-06 | GSE213125 | GEO

Epicatechin rescues memory deficits by inducing autophagy in tauopathy

Project description:Proteomics raw data for “Epicatechin rescues memory deficits by inducing autophagy in tauopathy”

2021-10-11 | PXD029068 |

Inhibition of TRPV4 rescues impaired excitability and social deficits in NAc-specific Shank3-deficient mice.

Project description:We used AAV transfection of Shank3 shRNA to study the effect of Shank3 knock down in Nucleus Accumbens neurons. We find that Shank3 knock down leads to social deficits and hyperexcitability of Nucleus Accumbens D1-MSN. Bulk RNA sequencing shows that D1-MSN with Shank3 knock down upregulate TRPV4 and its subsequent antagonist driven inhibition leads to both a rescue of D1-MSN hyperexcitability and social deficit.

2022-10-01 | GSE139683 | GEO

Retinoic acid supplementation rescues the social deficits in Fmr1 knockout mice

Project description:Retinoic acid supplementation rescues the social deficits in Fmr1 knockout mice

| PRJNA832607 | ENA

Butyrate, but not Propionate, Rescues Maternal Diet-Induced Neurocognitive Deficits in Offspring

Project description:Butyrate, but not Propionate, Rescues Maternal Diet-Induced Neurocognitive Deficits in Offspring

| PRJNA612803 | ENA

Single cell RNA-sequencing of polyA tailed RNA from mutant SOD1 and isogenic control human motor neurons

Project description:Amyotrophic Lateral Sclerosis is a fatal neurodegenerative disorder that affects motor neurons (MN). We used single cell RNA-seq of degenerating human MN derived from ALS patients to understand molecular drivers of MN degeneration. Patient-derived iPSC bearing a point mutation in the SOD1 gene (SOD1 E100G) were differentiated into MN. MN derived from CRISPR-Cas9 corrected isogenic control iPSC (SOD1 E100E) were used as control. Survival analysis indicated that at 44 days of in vitro differentiation, ALS MN dislpayed survival deficits. At this point, cells were harvested for single cell transcriptomics using the Fluidigm C1 system.

2019-11-01 | E-MTAB-7353 | biostudies-arrayexpress

VLDL Proteome in Control-AAV and PSS1(Q353R)-AAV

Project description:Fractionated VLDL from Control-AAV and PSS1(Q353R)-AAV.

2024-10-21 | PXD047916 | Pride

Inhibition of TRPV4 rescues impaired excitability and social deficits in NAc-specific Shank3-deficient mice.

Project description:Inhibition of TRPV4 rescues impaired excitability and social deficits in NAc-specific Shank3-deficient mice.

| PRJNA586875 | ENA

RNA sequencing approaches to identify the differential expression gene in the mPFC with AAV-mediated Npas4 shRNA expression

Project description:Chronic social defeat stress induces anhedonia-like behavior deficits. We demonstrated that mPFC NPAS is requred for chronic social defeat stress-induced deficits in the sucrose preference and effort-based reward seeking behavior. We conducted the RNA-seq analysis for differential expression genes in the mouse mPFC samples of AAV-mediated Npas4 shRNA expression tissue.

2023-02-10 | GSE165586 | GEO

Gene expression analysis of different motor neuron genotypes and their response to biological exposure and AAV treatment

Project description:Wild-type motor neurons and motor neurons carrying ALS-relevant mutations (TDP-43 - M337V; SOD1 - G93A) were cultured and exposed different biological treatments in the absence and presence of AAV(s) (1-5) transduction and consequent protein expression. Their response to these variables under different combinations was assessed with regards to gene expression using nCounter Gene Expression Panels (nanoString), Neuropathology and Neuroinflammation Panels.

2024-12-01 | GSE281698 | 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