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PXD031884

PXD031884 is an original dataset announced via ProteomeXchange.

Dataset Summary
TitleF8 intein precise CodopN6 reconstitution
DescriptionLiver 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 the development of circulating anti-F8 antibodies which neutralize F8 activity. Taking advantage of split inteins-mediated protein trans-splicing, we divided the coding sequence of the large and highly secreted F8-N6 variant in two separate AAV split-inteins vectors whose co-administration to HemA mice results in F8 protein reconstitution and expression of therapeutic levels of F8 over time without anti-F8 antibodies development.
HostingRepositoryPRIDE
AnnounceDate2022-11-16
AnnouncementXMLSubmission_2022-11-15_23:37:05.317.xml
DigitalObjectIdentifier
ReviewLevelPeer-reviewed dataset
DatasetOriginOriginal dataset
RepositorySupportUnsupported dataset by repository
PrimarySubmitterFedericaEsposito
SpeciesList scientific name: Homo sapiens (Human); NCBI TaxID: 9606;
ModificationListacetylated residue; iodoacetamide derivatized residue
InstrumentQ Exactive HF
Dataset History
RevisionDatetimeStatusChangeLog Entry
02022-02-24 10:27:25ID requested
12022-11-15 23:37:06announced
Publication List
Esposito F, Lyubenova H, Tornabene P, Auricchio S, Iuliano A, Nusco E, Merlin S, Olgasi C, Manni G, Gargaro M, Fallarino F, Follenzi A, Auricchio A, Liver gene therapy with intein-mediated F8 trans-splicing corrects mouse haemophilia A. EMBO Mol Med, 14(6):e15199(2022) [pubmed]
Keyword List
submitter keyword: protein trans-splicing, AAV-intein, haemophilia A, Liver gene therapy, full length protein reconstitution
Contact List
AlbertoAuricchio
contact affiliationTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy. Department of Advanced Biomedicine, Federico II University, Naples, Italy.
contact emailauricchio@tigem.it
lab head
FedericaEsposito
contact affiliationTelethon Institute of Genetics and Medicine (TIGEM)
contact emailf.esposito@tigem.it
dataset submitter
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Dataset FTP location
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