PXD031884 is an
original dataset announced via ProteomeXchange.
Dataset Summary
Title | F8 intein precise CodopN6 reconstitution |
Description | 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 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. |
HostingRepository | PRIDE |
AnnounceDate | 2022-11-16 |
AnnouncementXML | Submission_2022-11-15_23:37:05.317.xml |
DigitalObjectIdentifier | |
ReviewLevel | Peer-reviewed dataset |
DatasetOrigin | Original dataset |
RepositorySupport | Unsupported dataset by repository |
PrimarySubmitter | FedericaEsposito |
SpeciesList | scientific name: Homo sapiens (Human); NCBI TaxID: 9606; |
ModificationList | acetylated residue; iodoacetamide derivatized residue |
Instrument | Q Exactive HF |
Dataset History
Revision | Datetime | Status | ChangeLog Entry |
0 | 2022-02-24 10:27:25 | ID requested | |
⏵ 1 | 2022-11-15 23:37:06 | announced | |
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 affiliation | Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy. Department of Advanced Biomedicine, Federico II University, Naples, Italy. |
contact email | auricchio@tigem.it |
lab head | |
FedericaEsposito |
contact affiliation | Telethon Institute of Genetics and Medicine (TIGEM) |
contact email | f.esposito@tigem.it |
dataset submitter | |
Full Dataset Link List
Dataset FTP location
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PRIDE project URI |
Repository Record List
[ + ]
[ - ]
- PRIDE
- PXD031884
- Label: PRIDE project
- Name: F8 intein precise CodopN6 reconstitution