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ProteomicsRecombinant dimeric PICK1 protein inhibitors for long-term relief of chronic pain by AAV therapeutics
Chronic neuropathic pain’s impact, persistence, and limited treatments render it relevant for gene therapy. Here, we describe the development and application of self-assembling dimeric peptide inhibitors of the pain-associated scaffolding protein PICK1 (protein interacting with C-kinase 1) delivered...
ORGANISM(S): Mus musculus (Mouse) 
2026-04-10 | PXD074049 | Pride
Pick1  protein interacting with c-kinase 1  neuropathic pain Pain treatment  neuropathic pain treatment  gene therapy  aav therapeutics. 
ProteomicsRecombinant dimeric PICK1 protein inhibitors for long-term relief of chronic pain by AAV therapeutics
Chronic neuropathic pain’s impact, persistence, and limited treatments render it relevant for gene therapy. Here, we describe the development and application of self-assembling dimeric peptide inhibitors of the pain-associated scaffolding protein PICK1 (protein interacting with C-kinase 1) delivered...
ORGANISM(S): Mus musculus (Mouse) 
2026-04-10 | PXD073925 | Pride
Pick1  protein interacting with c-kinase 1  neuropathic pain Pain treatment  neuropathic pain treatment  gene therapy  aav therapeutics 
TranscriptomicsA multi-functional AAV-CRISPR-Cas9 and its host response
CRISPR-Cas9 delivery by AAV holds promise for gene therapy but faces critical barriers due to its potential immunogenicity and limited payload capacity. Here, we demonstrate genome engineering in postnatal mice using AAV-split-Cas9, a multi-functional platform customizable for genome-editing, transc...
ORGANISM(S): Mus musculus 
2016-07-29 | E-GEOD-84534 | biostudies-arrayexpress
UnknownEvolving AAV-delivered therapeutics towards ultimate cures.
Not available
S-EPMC7885987 | biostudies-literature
TranscriptomicsGenotoxicity after adeno-associated virus (AAV) gene therapy is dependent upon dose, treatment age and enhancer-promoter selection (microRNA)
AAV gene therapy has recently been approved for clinical use and shown to be efficacious and safe in a growing number of clinical trials. However, the safety of AAV as a gene therapy has been challenged by a few studies that documented hepatocellular carcinoma (HCC) after AAV gene delivery in mice. ...
ORGANISM(S): Mus musculus 
2015-09-01 | E-GEOD-61632 | biostudies-arrayexpress
TranscriptomicsTranscriptome analysis of cuprizone mouse model following AAV-mediated delivery of A Proliferation Inducing Ligand
A Proliferation Inducing Ligand (APRIL) is a member of the tumour necrosis factor (TNF) superfamily and has recently been shown to modulate pro-inflammatory astrocyte responses, as well as to ameliorate disease outcome in the experimental autoimmune encephalomyelitis mouse model for multiple scleros...
ORGANISM(S): Mus musculus 
2025-09-29 | E-MTAB-14516 | biostudies-arrayexpress
UnknownThe Effect of CpG Sequences on Capsid-Specific CD8+ T Cell Responses to AAV Vector Gene Transfer.
Not available
S-EPMC7054717 | biostudies-literature
UnknownA robust and flexible baculovirus-insect cell system for AAV vector production with improved yield, capsid ratios and potency.
Not available
S-EPMC10959708 | biostudies-literature
UnknownAAV-delivered PPT1 provides long-term neurological benefits in CLN1 mice and achieves therapeutic levels in sheep brain.
Not available
S-EPMC12848131 | biostudies-literature
UnknownMultiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.
Not available
S-EPMC8672712 | biostudies-literature
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