Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector

By M. Kay, C. Manno, M. Ragni, P. Larson, L. Couto, A. Mcclelland, B. Glader, A. Chew, S. J. Tai, R. Herzog and 6 more. Nature Genetics, 2000

1,049 citations recorded by the source corpus.

Subfields: Genetics, Molecular Biology, Oncology

View Paper: https://www.semanticscholar.org/paper/f3a5f9aef81cf97eb669e36cb157333a31d253db

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Citations in the corpus

Gene therapy comes of age (2018, neutral): “Pioneering AAV gene therapy clinical trials for hemophilia B were initiated in the late 1990s, first testing delivery of AAV vectors to muscle via injection (39) and then moving to intravenous administration, taking advantage of AAV2 liver tropism (40).”
Gene therapy: trials and tribulations (2000, background): “Current clinical trials with AAV vectors rely on transient production systems, which may be suitable for proof of principle, but a more efficient production system is urgently require”
Immune responses to AAV vectors: overcoming barriers to successful gene therapy. (2013, neutral): “The first trials of AAV vectors in human subjects were in the setting of cystic fibrosis(25); the first trial in hemophilia B, in which vector was injected into skeletal muscle, began in 1999.(26) A key concept in the study of immune responses triggered by in vivo gene transfer with AAV is that the ”
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response (2006, background): “IX delivered to skeletal muscle of individuals with severe hemophilia B was safe, but circulating levels were generally not sufficient to improve disease phenotyp”
Human and mouse proteases: a comparative genomic approach (2003, background): “Gene therapy, bone-marrow transplantation, enzyme enhancement therapies and substrate-deprivation strategies might offer therapeutic alternatives for human diseases that are caused by protease deficiencies, and in some cases, preliminary clinical trials have confirmed their potential effectiveness f”
Adeno-associated virus as a delivery vector for gene therapy of human diseases (2024, background): “In 2000, a phase I trial using i.m. delivery of rAAV. hFIX reported a favorable safety pro ﬁ le, 303 which was con ﬁ rmed three years later after the completion of the study.”
Design and development of polymers for gene delivery (2005, background): “reported positive data, including increased circulating levels of factor IX, in a haemophilia clinical tria”