Proteomics

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F8 intein precise CodopN6 reconstitution

ABSTRACT: 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.

INSTRUMENT(S): Q Exactive HF

ORGANISM(S): Homo Sapiens (human)

SUBMITTER: Federica Esposito  

LAB HEAD: Alberto Auricchio

PROVIDER: PXD031884 | Pride | 2022-03-31

REPOSITORIES: Pride

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Dataset's files

Source:
Action DRS
2020-06-26_MS_AA_FE_FVIIIinteins_codopN61st.raw Raw
2020-06-26_MS_AA_FE_FVIIIinteins_codopN62nd.raw Raw
2020-06-26_MS_AA_FE_FVIIIinteins_inteincodop1.raw Raw
2020-06-26_MS_AA_FE_FVIIIinteins_inteincodop2.raw Raw
2020-08-01_MS_AA_FE_CodopN6inteins_codop_A1.raw Raw
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Publications

Liver gene therapy with adeno-associated viral (AAV) vectors is under clinical investigation for haemophilia 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 neutralise F8 activity. Taking advantage of split-intein-mediated protein trans-splicing, we divided the coding sequence of the large and highly  ...[more]

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