Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

By C. Manno, V. Arruda, G. Pierce, B. Glader, M. Ragni, J. Rasko, Margareth C. Ozelo, K. Hoots, P. Blatt, B. Konkle and 20 more. Nature Medicine, 2006

2,132 citations recorded by the source corpus.

Subfields: Genetics, Molecular Biology, Oncology

View Paper: https://www.semanticscholar.org/paper/ee89113f47659dbfb46b0dc54f3858cc3bb29c0d

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In Genetics

1. C. June
2. K. High
3. B. Levine
4. F. Mingozzi
5. M. Kay
6. Guangping Gao
7. Wei Zhan
8. Thomas L. Gallagher

In Molecular Biology

1. K. High
2. N. Bertrand
3. F. Mingozzi
4. Xiaoyang Xu
5. O. Farokhzad
6. M. Kay
7. Guangping Gao
8. Wei Zhan

In Oncology

1. C. June
2. B. Levine
3. K. High
4. O. Farokhzad
5. M. Kay
6. J. Kochenderfer
7. Jennifer N. Brudno
8. Michel Sadelain

Citations in the corpus

• Immune Responses to Viral Gene Therapy Vectors (2020, background): “CD8 T Cell Responses to Capsid In the first liver-directed gene therapy trial with AAV vectors (performed with an AAV2 vector in patients with hemophilia B), a loss of factor IX transgene expression and transient mild elevations of liver enzymes in circulation were correlated with a CD8 T cell respo”
• Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B (2014, neutral): “5 13 202 58 (8) 7 35 12 70 6 13 36 69 (9) 5 2 8 54 7 29 43 55 (7) 2 2 8 0 8 13 17 47 (6) NA NA NA NA 9 17 20 63 (9) NA NA NA NA 10 32 64 66 (9) 7 7 8 50”
• Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors (2010, neutral): “Data indicate that even low levels of neutralizing antibodies (1:5–1:10) can completely abrogate transduction with high titers of vectors (Jiang et al. , 2006; Manno et al. , 2006; Scallan et al. , 2006).”
• Interactions of nanomaterials and biological systems: implications to personalized nanomedicine (2012, methodology): “This gene delivery vector, which is now in phase II/III for LCA, was developed in parallel with an analogous formulation containing encoding DNA for the human coagulation factor IX, for the treatment of hemophilia B [230].”
• Immune responses to AAV vectors: overcoming barriers to successful gene therapy. (2013, positive): “15,39 Use empty capsids to adsorb anti-AAV antibodies, thus allowing for vector transduction.”
• Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates (2009, background): “The use of viral vectors for gene therapy has been thwarted by immune responses to the viral vector particles and/or the transduced target cells, leading to transient and acute toxicity that precluded long-term expression of the potentially therapeutic gene product [22].”
• Emerging Issues in AAV-Mediated In Vivo Gene Therapy (2017, background): “However, this is not an ideal solution to the problem because not all individuals may have an immune response to the vector, and the timing of immune responses may vary according to the vector dose and serotype.(48,49,57,69) Then immunosuppression performed at the time of AAV injection may change th”