Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy

Etranacogene dezaparvovec (AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing a codon-optimized Padua variant human factor IX (FIX) transgene with a liver-specific promoter. Here, we report 3-year outcomes from a phase 2b, open-label, single-dose, single-arm, multic...

Descripción completa

Detalles Bibliográficos
Autores principales: von Drygalski, Annette, Gomez, Esteban, Giermasz, Adam, Castaman, Giancarlo, Key, Nigel S., Lattimore, Susan U., Leebeek, Frank W. G., Miesbach, Wolfgang A., Recht, Michael, Gut, Robert, Dolmetsch, Ricardo, Monahan, Paul E., Le Quellec, Sandra, Pipe, Steven W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The American Society of Hematology 2022
Materias:
Gene Therapy
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10539871/
https://www.ncbi.nlm.nih.gov/pubmed/36490302
http://dx.doi.org/10.1182/bloodadvances.2022008886

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10539871/
https://www.ncbi.nlm.nih.gov/pubmed/36490302
http://dx.doi.org/10.1182/bloodadvances.2022008886

Ejemplares similares