Browse
Submit Data
Databases
API
Help

Dataset Information

0 Views

0 Connections

0 Citations

0 Reanalyses

0 Downloads

Omics score: 0

RAAV-miniBEND: A Targeted Vector for Brain Endothelial Cell Gene Delivery and Cerebrovascular Malformation Modeling

ABSTRACT: rAAV-miniBEND: A Targeted Vector for Brain Endothelial Cell Gene Delivery and Cerebrovascular Malformation Modeling

PROVIDER: PRJNA1041312 | ENA |

REPOSITORIES: ENA

ACCESS DATA

Json Xml

Similar Datasets

rAAV-miniBEND: A Targeted Vector for Brain Endothelial Cell Gene Delivery and Cerebrovascular Malformation Modeling II

Project description:rAAV-miniBEND: A Targeted Vector for Brain Endothelial Cell Gene Delivery and Cerebrovascular Malformation Modeling II

| PRJNA1117954 | ENA

rAAV-miniBEND: A Targeted Vector for Brain Endothelial Cell Gene Delivery and Cerebrovascular Malformation Modeling

Project description:In-depth analysis of the molecular mechanisms underlying the initiation and development of bAVM was conducted through single-nucleus RNA sequencing of cells from bAVM lesions in Braf-CAfl/fl mice locally injected with AAV-miniBEND.

2025-04-29 | GSE247963 | GEO

rAAV-miniBEND: A Targeted Vector for Brain Endothelial Cell Gene Delivery and Cerebrovascular Malformation Modeling II

2025-04-29 | GSE268643 | GEO

Support-vector-machine learning analysis of the human cerebrovascular thrombi proteome: an exploratory study

Project description:Mechanical retrieval of thrombotic material from acute ischemic stroke subjects provides a unique entry point for translational research investigations. Here, we resolved the proteomes of cardioembolic and atherothrombotic cerebrovascular human thrombi and applied an articial intelligence routine to analyze potential protein signatures between the two selected groups.

2020-11-05 | PXD020398 | Pride

Project description:Intrathymic delivery of rAAV

| PRJNA832955 | ENA

Design of a novel highly efficient peptide-based siRNA-delivery vector, PepFect6

Project description:While small interfering RNAs (siRNAs) have been rapidly appreciated to induce gene silencing, efficient vectors for primary cells and for systemic in vivo delivery are lacking. We here present a novel chemically modified cell-penetrating peptide named PepFect6 (PF6) for efficient delivery of siRNAs into various types of cells including e.g. lymphocyte suspension cells and primary embryonic stem cells. Stable PF6/siRNA nano- particles rapidly enter entire cell populations resulting in strong and persistent RNAi responses, without associated transcriptomic or proteomic changes. In contrast to the majority of chemical reagents, PF6-mediated delivery is independent of cell confluence and, in most cases, not significantly hampered by the presence of serum. Finally, strong RNAi responses are observed in two different in vivo models following systemic delivery of PF6/siRNA in mice. Taken together, PF6 is an efficient siRNA delivery vector with superior delivery properties as compared to other tested transfection reagents.

2009-12-31 | GSE19702 | GEO

Production of rAAV by plasmid transfection induces antiviral and inflammatory responses in suspension HEK293

Project description:To understand the response of host cells to rAAV vector production via plasmid transfection, samples were collected throughout the course of three 50 L production batches and analyzed by bulk RNA sequencing of polyadenylated transcripts (RNA-seq). Gene ontology analysis determined that upregulated pathways included inflammatory and antiviral responses. Systematic analyses of the cellular transcriptional response to rAAV production indicates that these host cells are not passively supporting vector manufacture, and therefore may illuminate genes and pathways that influence rAAV production, thereby enabling the rational design of next-generation manufacturing platforms to support safe, effective, and affordable AAV-based gene therapies.

2023-02-06 | GSE224405 | GEO

Correlation of antigen expression with epigenetic modifications after rAAV delivery of a human Factor IX variant in mice and rhesus macaques (Tn5 mouse)

Project description:We investigated long-term human coagulation Factor IX expression of a novel variant when delivered into mice and rhesus macaques and compare transduction efficiencies using two different AAV capsids. In hemophilic mice injected with KP1-packaged rAAV expressing the hyperactive FIX variant specific activity plasma levels were 10-fold or 2-fold enhanced when compared to wild-type or Padua huFIX injected mice, respectively. In rhesus macaques AAV-LK03 capsid outperformed AAV-KP1 in terms of antigen expression and liver transduction. Two animals from each group showed sustained low-level huFIX expression at 3 months post-administration, while one animal from each group lost huFIX mRNA and protein expression over time despite comparable vector copies. We investigated if epigenetic differences in the vector episomes could explain this loss of transcription. Cut&Tag analysis revealed lower levels of activating histone marks in the two animals that lost expression. When comparing rAAV genome associated histone modifications in rhesus macaques to those in mice injected with the same vector, the activating histone marks were starkly reduced in macaque-derived episomes. Differential epigenetic marking of AAV genomes may explain different expression profiles in mice and rhesus macaques as well as the wide dose response variation observed in primates in both preclinical and human clinical trials.

2024-03-11 | GSE261045 | GEO

Correlation of antigen expression with epigenetic modifications after rAAV delivery of a human Factor IX variant in mice and rhesus macaques (CnT NHP)

2024-03-11 | GSE261044 | GEO

Correlation of antigen expression with epigenetic modifications after rAAV delivery of a human Factor IX variant in mice and rhesus macaques (Tn5 NHP)

2024-03-11 | GSE261046 | GEO