
NCBI . NLM . NIH . GOV {
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Title[redir]:
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A - PMC
Description:
The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose. In this phase 1β2 trial, we infused an ...
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Keywords {π}
factor, viii, participants, expression, vector, hemophilia, gene, activity, spk, administration, google, scholar, immune, response, doi, aav, pubmed, bleeding, events, trial, normal, data, pmc, therapy, weeks, article, kilogram, range, received, glucocorticoids, capsid, participant, safety, assay, free, transfer, year, med, efficacy, results, adverse, onestage, annualized, infusion, smf, dose, event, rate, fig, determined,
Topics {βοΈ}
b-domainβdeleted form aav5-hfviii-sq gene therapy glucocorticoid-sparing immune-modulating agents hepatocyte-derived factor viii aav5-hfviii-sq differs plasma-derived factor viii highest-dose cohort showed liver-directed gene transfer aav-mediated gene transfer pmc beta search protein-engineered coagulation factors aav-mediated gene therapy cross-reacting material supplementary materials included pmc copyright notice aav5-hfviii-sq peripheral-blood mononuclear cells baculovirus vector-production system time disease-altering therapy aav-factor ix recombinant-aav vector consisting durable hepatocyte expression coagulation factor viii 4-year long-term follow plasma factor viii factor ix consumption ehl extended half-life drug-related adverse events mediated gene transfer factor viii activity lowest-dose cohort received factor viii function bioengineered capsid derived immune-modulating agents aav5-hfviii-sq7 factor viii expression vector-dose dependent factor viii product factor viii infusions recombinant factor viii factor viii padua glucocorticoid-sparing agents supplementary methods section [google scholar] 12 [google scholar] 20 [google scholar] 23 recurrent joint hemorrhage durable therapeutic efficacy stable fix expression aav gene transfer
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