References
-
Naso MF, Tomkowicz B, Perry WL 3rd, Strohl WR. Adeno-associated virus (AAV) as a vector for gene therapy. BioDrugs. 2017;31:317–34.
-
Bulcha JT, Wang Y, Ma H, Tai PWL, Gao G. Viral vector platforms within the gene therapy landscape. Signal Transduct Target Ther. 2021;6:53.
-
Nidetz NF, McGee MC, Tse LV, Li C, Cong L, Li Y, et al. Adeno-associated viral vector-mediated immune responses: understanding barriers to gene delivery. Pharmacol Ther. 2020;207:107453.
-
Leborgne C, Latournerie V, Boutin S, Desgue D, Quere A, Pignot E, et al. Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients. Cell Immunol. 2019;342:103780.
-
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med. 2006;12:342–7.
-
Boutin S, Monteilhet V, Veron P, Leborgne C, Benveniste O, Montus MF, et al. 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. Hum Gene Ther. 2010;21:704–12.
-
Calcedo R, Wilson JM. Humoral immune response to AAV. Front Immunol. 2013;4:341.
-
Gorovits B, Azadeh M, Buchlis G, Fiscella M, Harrison T, Havert M, et al. Evaluation of cellular immune response to adeno-associated virus-based gene therapy. AAPS J. 2023;25:47.
-
Verdera HC, Kuranda K, Mingozzi F. AAV vector immunogenicity in humans: a long journey to successful gene transfer. Mol Ther. 2020;28:723–46.
-
Calcedo R, Vandenberghe LH, Gao G, Lin J, Wilson JM. Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis. 2009;199:381–90.
-
Greenberg B, Butler J, Felker GM, Ponikowski P, Voors AA, Pogoda JM, et al. Prevalence of AAV1 neutralizing antibodies and consequences for a clinical trial of gene transfer for advanced heart failure. Gene Ther. 2016;23:313–9.
-
Khatri A, Shelke R, Guan S, Somanathan S. Higher seroprevalence of anti-adeno-associated viral vector neutralizing antibodies among racial minorities in the United States. Hum Gene Ther. 2022;33:442–50.
-
Erles K, Sebökovà P, Schlehofer JR. Update on the prevalence of serum antibodies (IgG and IgM) to adeno-associated virus (AAV). J Med Virol. 1999;59:406–11.
-
Moskalenko M, Chen L, van Roey M, Donahue BA, Snyder RO, McArthur JG, et al. Epitope mapping of human anti-adeno-associated virus type 2 neutralizing antibodies: implications for gene therapy and virus structure. J Virol. 2000;74:1761–6.
-
Mendell JR, Connolly AM, Lehman KJ, Griffin DA, Khan SZ, Dharia SD, et al. Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations. Mol Ther Methods Clin Dev. 2022;25:74–83.
-
Chhabra A, Bashirians G, Petropoulos CJ, Wrin T, Paliwal Y, Henstock PV, et al. Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies. Mol Ther Methods Clin Dev. 2024;32. https://doi.org/10.1016/j.omtm.2024.101273.
-
Schulz M, Levy DI, Petropoulos CJ, Bashirians G, Winburn I, Mahn M, et al. Binding and neutralizing anti-AAV antibodies: detection and implications for rAAV-mediated gene therapy. Mol Ther. 2023;31:616–30.
-
Verma S, Nwosu SN, Razdan R, Upadhyayula SR, Phan HC, Koroma AA, et al. Seroprevalence of adeno-associated virus neutralizing antibodies in males with Duchenne muscular dystrophy. Hum Gene Ther. 2023;34:430–8.
-
Weber T. Anti-AAV antibodies in AAV gene therapy: current challenges and possible solutions. Front Immunol. 2021;12:658399.
-
Jiang H, Couto LB, Patarroyo-White S, Liu T, Nagy D, Vargas JA, et al. Effects of transient immunosuppression on adeno-associated virus-mediated liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood. 2006;108:3321–8.
-
Smith CJ, Ross N, Kamal A, Kim KY, Kropf E, Deschatelets P, et al. Pre-existing humoral immunity and complement pathway contribute to immunogenicity of adeno-associated virus (AAV) vector in human blood. Front Immunol. 2022;13. https://doi.org/10.3389/fimmu.2022.999021.
-
Arjomandnejad M, Dasgupta I, Flotte TR, Keeler AM. Immunogenicity of recombinant adeno-associated virus (AAV) vectors for gene transfer. BioDrugs. 2023;37:311–29.
-
Klamroth R, Hayes G, Andreeva T, Gregg K, Suzuki T, Mitha IH, et al. Global seroprevalence of pre-existing immunity against AAV5 and other AAV serotypes in people with Hemophilia A. Hum Gene Ther. 2022;33:432–41.
-
Real Decreto 1088/2005, de 16 de septiembre, por el que se establecen los requisitos técnicos y condiciones mínimas de la hemodonación y de los centros y servicios de transfusión. Boletín Oficial del Estado núm.2005;225. Accessed at: https://www.sanidad.gob.es/profesionales/saludPublica/medicinaTransfusional/legislacion/docs/RD_1088-2005.pdf.
-
Lock M, Alvira M, Vandenberghe LH, Samanta A, Toelen J, Debyser Z, et al. Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum Gene Ther. 2010;21:1259–71.
-
Piedra J, Ontiveros M, Miravet S, Penalva C, Monfar M, Chillon M. Development of a rapid, robust, and universal picogreen-based method to titer adeno-associated vectors. Hum Gene Ther Methods. 2015;26:35–42.
-
Thwaite R, Pagès G, Chillón M, Bosch A. AAVrh.10 immunogenicity in mice and humans. Relevance of antibody cross-reactivity in human gene therapy. Gene Ther. 2014;22:196–201.
-
O’Connor DM, Lutomski C, Jarrold MF, Boulis NM, Donsante A. Lot-to-Lot variation in adeno-associated virus serotype 9 (AAV9) preparations. Hum Gene Ther Methods. 2019;30:214–25.
-
Ling C, Wang Y, Feng YL, Zhang YN, Li J, Hu XR, et al. Prevalence of neutralizing antibodies against liver-tropic adeno-associated virus serotype vectors in 100 healthy Chinese and their potential relation to body constitutions. J Integr Med. 2015;13:341–6.
-
Liu Q, Huang W, Zhang H, Wang Y, Zhao J, Song A, et al. Neutralizing antibodies against AAV2, AAV5 and AAV8 in healthy and HIV-1-infected subjects in China: implications for gene therapy using AAV vectors. Gene Ther. 2014;21:732–8.
-
Mingozzi F, High KA. Immune responses to AAV vectors: overcoming barriers to successful gene therapy. Blood. 2013;122:23–36.
-
Calcedo R, Morizono H, Wang L, McCarter R, He J, Jones D, et al. Adeno-associated virus antibody profiles in newborns, children, and adolescents. Clin Vaccin Immunol. 2011;18:1586–8.
-
Kruzik A, Fetahagic D, Hartlieb B, Dorn S, Koppensteiner H, Horling FM, et al. Prevalence of anti-adeno-associated virus immune responses in international cohorts of healthy donors. Mol Ther Methods Clin Dev. 2019;14:126–33.
-
Mingozzi F, Chen Y, Edmonson SC, Zhou S, Thurlings RM, Tak PP, et al. Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue. Gene Ther. 2013;20:417–24.
-
Navarro-Oliveros M, Vidaurrazaga A, Soares Guerra G, Castellana D, Embade N, Millet O, et al. Seroprevalence of adeno-associated virus types 1, 2, 3, 4, 5, 6, 8, and 9 in a Basque cohort of healthy donors. Sci Rep. 2024;14:15941.
-
Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, Zhou X, et al. Clades of Adeno-associated viruses are widely disseminated in human tissues. J Virol. 2004;78:6381–8.
-
Halbert CL, Miller AD, McNamara S, Emerson J, Gibson RL, Ramsey B, et al. Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: implications for gene therapy using AAV vectors. Hum Gene Ther. 2006;17:440–7.
-
Gardner MR, Mendes DE, Muniz CP, Martinez-Navio JM, Fuchs SP, Gao G, et al. High concordance of ELISA and neutralization assays allows for the detection of antibodies to individual AAV serotypes. Mol Ther Methods Clin Dev. 2022;24:199–206.
-
Dhungel BP, Winburn I, Pereira CDF, Huang K, Chhabra A, Rasko JEJ. Understanding AAV vector immunogenicity: from particle to patient. Theranostics. 2024;14:1260–88.
-
Braun M, Lange C, Schatz P, Long B, Stanta J, Gorovits B, et al. Preexisting antibody assays for gene therapy: considerations on patient selection cutoffs and companion diagnostic requirements. Mol Ther Methods Clin Dev. 2024;32:101217.
-
Li C, Narkbunnam N, Samulski RJ, Asokan A, Hu G, Jacobson LJ, et al. Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia. Gene Ther. 2012;19:288–94.
-
Pabinger I, Ayash-Rashkovsky M, Escobar M, Konkle BA, Mingot-Castellano ME, Mullins ES, et al. Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia. Gene Ther. 2024;31:273–84.
-
Falese L, Sandza K, Yates B, Triffault S, Gangar S, Long B, et al. Strategy to detect pre-existing immunity to AAV gene therapy. Gene Ther. 2017;24:768–78.
-
Gorovits B, Fiscella M, Havert M, Koren E, Long B, Milton M, et al. Recommendations for the development of cell-based anti-viral vector neutralizing antibody assays. AAPS J. 2020;22:24.
