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ProteomicsF8 intein precise CodopN6 reconstitution
Liver gene therapy with adeno-associated viral (AAV) vectors is under clinical investigation for hemophilia A (HemA), the most common inherited X-linked bleeding disorder. Major limitations are the large size of the F8 transgene, which makes packaging in a single AAV vector a challenge, as well as t...
ORGANISM(S): Homo sapiens (Human) 
2022-03-31 | PXD031884 | Pride
Protein trans-splicing Aav-intein Haemophilia a Liver gene therapy Full length protein reconstitution 
TranscriptomicsGenetic analysis reveals a longevity associated protein modulating endothelial function and angiogenesis
Transcriptome analysis in HEK293T transfected with plasmid carrying different isoforms of BPIFB4 gene. This gene was previously associated with exceptional longevity in a GWAS study performed on three different populations. Results indicate an up-regulation of stress response genes and proteostasis ...
ORGANISM(S): Homo sapiens 
2015-06-12 | E-GEOD-63912 | biostudies-arrayexpress
UnknownLiver gene therapy with intein-mediated F8 trans-splicing corrects mouse hemophilia A
Not available
S-SCDT-EMM-2021-15199 | biostudies-other
UnknownLiver-directed gene therapy for ornithine aminotransferase deficiency.
Not available
S-SCDT-10_15252-EMMM_202217033 | biostudies-other
UnknownLight-responsive microRNA miR-211 targets Ezrin to modulate lysosomal biogenesis and retinal cell clearance
Not available
S-SCDT-EMBOJ-2019-102468 | biostudies-other
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