Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy

Day, John; R. Mendell, Jerry; H.M. Burghes, Arthur; W. van Olden, Rudolf; Adhikary, Rishi; W. Dilly, Keith

doi:10.1016/j.omtm.2023.101117

Public

Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy

Published in Molecular Therapy — Methods & Clinical Development • Sep 20, 2023

Authors:

John Day

Jerry R. Mendell

Arthur H.M. Burghes

Abstract

Onasemnogene abeparvovec is a recombinant adeno-associated virus serotype 9 (AAV9) vector-based gene therapy for spinal muscular atrophy (SMA). Patients with elevated titers of anti-AAV9 antibodies (AAV9-Ab) should not receive onasemnogene abeparvovec because of potential safety and efficacy implica...

Subject

Seroprevalence

Titer

Medicine

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