Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A.

K. John Pasi,Savita Rangarajan,Nina Mitchell,Will Lester,Emily Symington,Bella Madan,Michael Laffan,Chris B. Russell,Mingjin Li,Glenn F. Pierce,Wing Yen Wong

Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A.

2020

K. John Pasi
Savita Rangarajan
Nina Mitchell
Will Lester
Emily Symington
Bella Madan
Michael Laffan
Chris B. Russell
Mingjin Li
Glenn F. Pierce
Wing Yen Wong

Abstract Background Adeno-associated virus (AAV)–mediated gene therapy is under investigation as a therapeutic option for persons with hemophilia A. Efficacy and safety data include 3 years of foll...

Keywords:

Genetic enhancement
Surgery
Virus
MEDLINE
Diabetes mellitus
Internal medicine
Medicine
Biomarker (medicine)
follow up studies
Young adult
Clinical trial

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