Evaluation of the Humoral Response to Adeno-Associated Virus-Based Gene Therapy Modalities Using Total Antibody Assays-Reference-Cited by-同舟云学术

Evaluation of the Humoral Response to Adeno-Associated Virus-Based Gene Therapy Modalities Using Total Antibody Assays

Published:2021-09-16 Issue:6 Volume:23 Page:
ISSN:1550-7416
Container-title:The AAPS Journal
language:en
Short-container-title:AAPS J

Author:

Gorovits Boris^ORCID,Azadeh Mitra,Buchlis George,Harrison Travis,Havert Mike,Jawa Vibha,Long Brian,McNally Jim,Milton Mark,Nelson Robert,O’Dell Mark,Richards Karen,Vettermann Christian,Wu Bonnie

Publisher

Springer Science and Business Media LLC

Subject

Pharmaceutical Science

Link

https://link.springer.com/content/pdf/10.1208/s12248-021-00628-3.pdf

Reference79 articles.

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2. D'Avola D, López-Franco E, Sangro B, Pañeda A, Grossios N, Gil-Farina I, et al. Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria. J Hepatol. 2016;65(4):776–83. https://doi.org/10.1016/j.jhep.2016.05.012.

3. Miesbach W, Meijer K, Coppens M, Kampmann P, Klamroth R, Schutgens R, et al. Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B. Blood. 2018;131(9):1022–31. https://doi.org/10.1182/blood-2017-09-804419.

4. Long BR, Veron P, Kuranda K, Hardet R, Mitchell N, Hayes GM, et al. Early phase clinical immunogenicity of valoctocogene roxaparvovec, an AAV5-mediated gene therapy for hemophilia A. Molecular Therapy : the Journal of the American Society of Gene Therapy. 2021;29(2):597–610. https://doi.org/10.1016/j.ymthe.2020.12.008.

5. Long BR, Sandza K, Holcomb J, Crockett L, Hayes GM, Arens J, et al. The impact of pre-existing immunity on the non-clinical pharmacodynamics of AAV5-based gene therapy. Mol Ther Methods Clin Dev. 2019;13:440–52. https://doi.org/10.1016/j.omtm.2019.03.006.

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