sAAVient Therapeutics publications relevant to:

NextGen AAV vectors

  1. L. Zhong, B. Li, C.S. Mah, L. Govindasamy, M. Agbandje-McKenna, M.A. Cooper, R.W. Herzog, I. Zolotukhin, K.H. Warrington, Jr., K.A. Weigel-Van Aken, J.A. Hobbs, S. Zolotukhin, N. Muzyczka, and A. Srivastava. Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at reduced doses. Proc. Natl. Acad. Sci., USA, 105: 7827-7832, 2008.
  2. D. Markusic, R.W. Herzog, G. Aslanidi, B. Hoffman, B. Li, M. Li, G.R. Jayandharan, C. Ling, I. Zolotukhin, W. Ma, S. Zolotukhin, A. Srivastava, and L. Zhong. High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines. Mol. Therapy, 18: 2048-2056, 2010.
  3. G.V. Aslanidi, A.E. Rivers, L. Ortiz, L. Govindasamy, C. Ling, G.R. Jayandharan, S. Zolotukhin, M. Agbandje-McKenna, and A. Srivastava. High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors. Vaccine, 30: 3908-3917, 2012.
  4. A.T. Martino, E. Basner-Tschakarjan, D.M. Markusic, J. Finn, C. Hinderer, S. Zhou, D.A. Ostrov, A. Srivastava, H.C.J. Ertl, C. Terhorst, K.A. High, F. Mingozzi, and R.W. Herzog. Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells. Blood, 121: 2224-33, 2013.
  5. G.V. Aslanidi, A.E. Rivers, L. Ortiz, C. Ling, L. Song, L. Govindasamy, M. Tan, M. Agbandje-McKenna, and A. Srivastava. Optimization of recombinant AAV2 vectors for gene therapy: The final threshold? PLoS One, 8(3): e59142, 2013.
  6. B. Li, W. Ma, G.V. Aslanidi, C. Ling, K.V. Vliet, L.-y. Huang, M. Agbandje-McKenna, A. Srivastava, and G.A. Aslanidi. Site-directed mutagenesis of surface-exposed lysine residues leads to high-efficiency transduction by recombinant AAV2, but not AAV8 vectors. Hum. Gene Therapy Methods, 26: 211-220, 2015.

GenX AAV vectors

GenY AAV Vectors

GenZ AAV Vectors

Opt AAV Vectors

  1. C. Ling, B. Li, W. Ma, and A. Srivastava. Development optimized AAV serotype vectors for high-efficiency transduction at further reduced doses. Hum. Gene Ther. Meth., 27: 143-149, 2016.
  2. J. Shoti, K. Qing, G.D. Keeler, D. Duan, B.J. Byrne, and A. Srivastava. Development of capsid-and genome-modified optimized AAVrh74 vectors for muscle gene therapy. Mol. Ther. Meth. Clin. Dev., 31: 101147, 2023.

Synthetic AAV Vectors

  1. P. Nahreini, S.H. Larsen, and A. Srivastava. Cloning and integration of DNA fragments in human cells via the inverted terminal repeats o the adeno-associated virus 2 genome. Gene, 119: 265-272, 1992.
  2. J. Shoti, K. Qing, and A. Srivastava. Development of an AAV DNA-based synthetic vector for the potential gene therapy of hemophilia in children. Front. Microbiol., 13: 1033615, 2022.

Liver-tropism

  1. S. Ponnazhagan, P. Mukherjee, M.C. Yoder, X.-S. Wang, S.Z. Zhou, J. Kaplan, S. Wadsworth and A. Srivastava. Adeno-associated virus 2-mediated gene transfer in vivo: Organ-tropism and expression of transduced sequences in mice. Gene, 190: 203-210, 1997.
  2. L. Zhong, W. Li, Z, Yang, L.Y. Chen, Y. Li, K.Y. Qing, K.A. Weigel-Kelley, M.C. Yoder, W. Shou and A. Srivastava. Improved transduction of primary murine hepatocytes by recombinant adeno-associated virus 2 vectors in vivo. Gene Therapy, 11: 1165-1169, 2004.
  3. L.G. Glushakova, M.J. Lisankie, E.B. Eruslanov, C. Ojano-Dirain, I. Zolotukhin, L. Zhong, C. Liu, A. Srivastava, and P.W. Stacpoole. AAV3-mediated transfer and expression of the pyruvate dehydrogenase E1a subunit gene causes metabolic remodeling and apoptosis in human liver cancer cells. Mol. Genet. Metabol., 98: 289-299, 2009
  4. C. Ling, Y. Lu, J. Kalsi, G.R. Jayandharan, B. Li, W. Ma, B. Cheng, S. Gee, K. McGoogan, L. Zhong, L. Govindasamy, M. Agbandje-McKenna, and A. Srivastava. Hepatocyte growth factor receptor is a cellular coreceptor for adeno-associated virus 3. Hum. Gene Therapy, 21: 1741-1747, 2010.
  5. G.R. Jayandharan, L. Zhong, B.K. Sack, A.E. Rivers, M. Li, B. Li, R.W. Herzog, and A. Srivastava. Optimized adeno-associated virus (AAV)-protein phosphatase-5 helper viruses for efficient liver transduction by single-stranded AAV vectors: Therapeutic expression of Factor IX at reduced vector doses. Hum. Gene Therapy, 21: 271-283, 2010.
  6. Ling, Y. Lu, B. Cheng, K.E. McGoogan, S.W.Y. Gee, W. Ma, B. Li, G.V. Aslanidi, and A. Srivastava. High-efficiency transduction of liver cancer cells by recombinant adeno-associated virus serotype 3 vectors. J. Vis. Exp., 49. Pii: 2538, doi: 10.3791/2538, 2011.
  7. B. Cheng, C. Ling, Y. Dai, L.G. Glushakova, Y. Lu, S.W.Y. Gee, K.E. McGoogan, G.V. Aslanidi, M. Park, P.W. Stacpoole, D. Siemann, C. Liu, A. Srivastava, and C. Ling. Development of optimized AAV3 serotype vectors: Mechanism of high-efficiency transduction of human liver cancer cells. Gene Therapy, 19: 375-384, 2012.
  8. C. Ling, Y. Wang, Y. Zhang, A. Ejjigani, Z. Yin, Y. Lu, L. Wang, M. Wang, J. Li, Z. Hu, G.V. Aslanidi, L. Zhong, G. Gao, A. Srivastava, and C. Ling. Selective in vivo targeting of human liver tumors by optimized recombinant AAV3 vectors in a murine xenograft model. Human Gene Therapy, 25: 1023-1034, 2014.
  9. S. Li, C. Ling, L. Zhong, M. Li, Q. Su R. He, Q. Tang, D.L. Greiner, L.D. Shultz, M.A. Brehm, T.R. Flotte, C. Mueller, A. Srivastava, and G. Gao. Efficient and targeted transduction of non-human primate liver with systemically delivered optimized AAV3B vectors. Mol. Therapy, 23: 1867-1876, 2015.
  10. K. Vercauteren, B.E. Hoffman, I. Zolotukhin, J.W. Xiao, E. Basner-Tshakarjan, K.A. High, H.C.J. Ertl, C.M. Rice, A. Srivastava, Y.P. de Jong, and R.W. Herzog. Superior in vivo transduction of human hepatocytes using engineered AAV3 capsid. Mol. Therapy, 24: 1042-1049, 2016.
  11. D. Markusic, T.C. Nichols E.P. Merricks, B. Palaschak, I. Zolotukhin; D. Marsic; S. Zolotukhin, A. Srivastava, and R.W. Herzog. Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models. J. Transl. Medicine, 15: 94, 2017.
  12. H.C. Brown, C.B. Doering, R.W. Herzog, C. Ling, D.M. Markusic, H.T. Spencer, A. Srivastava, and A. Srivastava. Development of a clinical candidate AAV3 vector for gene therapy of hemophilia B. Hum. Gene Therapy, 31: 1114-1123, 2020.
  13. S.R.P. Kumar, J. Xie, S. Hu, J. Ko, Q. Huang, H.C. Brown, A. Srivastava, D.M. Markusic, C.B. Doering, H.T. Spencer, A. Srivastava, G. Gao, and R.W. Herzog. Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette. Mol. Ther Meth. Clin. Devel., 23: 98-107, 2021.
  14. L. Yin, G.D. Keeler, Y. Zhang, B.E. Hoffman, C. Ling, K. Qing, and A. Srivastava. AAV3-miRNA vectors for growth suppression of human hepatocellular carcinoma cells in vitro and human liver tumors in a murine xenograft model in vivo. Gene Therapy, 28: 422-434, 2021.
  15. H.D. Daniel, S. Kumar, R. Kannangai, K.M. Lakshmi, M. Agbandje-Mckenna, K. Coleman K, A. Srivastava, A. Srivastava, and A.M. Abraham. Prevalence of adeno-associated virus 3 capsid binding and neutralizing antibodies in healthy and hemophilia B individuals from India. Hum. Gene Therapy, 32: 451-457, 2021.
  16. H.D. Daniel, S. Kumar, R. Kannangai, J. Farzana, J.F. Noel, A. Abraham, K.M. Lakshmi, M. Agbandje-Mckenna, K. Coleman K, A. Srivastava, A. Srivastava, and A.M. Abraham. Age-stratified adeno-associated virus serotype 3 neutralizing and total antibody prevalence in hemophilia A patients from India. J. Med. Virology, 94: 4542-4547, 2022.

Supporting publications

  1. K.Y. Qing, X.-S. Wang, D.M. Kube, S. Ponnazhagan, A. Bajpai and A. Srivastava. Role of tyrosine phosphorylation of a cellular protein in adeno-associated virus 2-mediated transgene expression. Proc. Natl. Acad. Sci., USA, 94: 10879-10884, 1997.
  2. X.-S. Wang, K.Y. Qing, S. Ponnazhagan and A. Srivastava. Adeno-associated virus 2 DNA replication in vivo: Mutation analyses of the D-sequence in viral inverted terminal repeats. J. Virology, 71: 3077-3082, 1997.
  3. C. Mah, K.Y. Qing, B. Khuntirat, S. Ponnazhagan, X.-S. Wang, D.M. Kube, M.C. Yoder and A. Srivastava. Adeno-associated virus 2-mediated gene transfer: Role of epidermal growth factor receptor protein tyrosine kinase in transgene expression. J. Virology, 72: 9835-9843, 1998.
  4. K.Y. Qing, B. Khuntirat, C. Mah, D.M. Kube, X.-S. Wang, S. Ponnazhagan, S.Z. Zhou, V.J. Dwarki, M.C. Yoder and A. Srivastava. Adeno-associated virus type 2-mediated gene transfer: Correlation of tyrosine phosphorylation of the cellular single-stranded D sequence-binding protein with transgene expression in human cells in vitro and murine tissues in vivo. J. Virology, 72: 1593-1599, 1998.
  5. K.Y. Qing, C. Mah, J. Hansen, S.Z. Zhou, V.J. Dwarki and A. Srivastava. Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nature Medicine, 5: 71-77, 1999.
  6. K.Y. Qing, J. Hansen, K.A. Weigel-Kelley, M.Q. Tan, S.Z. Zhou and A. Srivastava. Adeno-associated virus 2-mediated gene transfer: Role of cellular FKBP52 protein in transgene expression. J. Virology, 75: 8968-8976, 2001.
  7. K.Y. Qing, W. Li, L. Zhong, M.Q. Tan, J. Hansen, K.A. Weigel-Kelley, L. Chen, M.C. Yoder and A. Srivastava. Adeno-associated virus 2-mediated gene transfer: Role of T-cell protein tyrosine phosphatase in transgene expression in established cell lines in vitro and transgenic mice in vivo. J. Virology, 77: 2741-2746, 2003.
  8. L. Zhong, L.Y. Chen, Y. Li, K.Y. Qing, K.A. Weigel-Kelley, R.J. Chan, M.C. Yoder, and A. Srivastava. Self-complementary adeno-associated virus 2 (AAV)-T cell protein tyrosine phosphatase vectors as helper-viruses to improve transduction efficiency of conventional single-stranded AAV vectors in vitro and in vivo. Mol. Therapy, 10: 950-957, 2004.
  9. L. Zhong, K.Y. Qing, Y. Si, L. Chen, M.Q. Tan and A. Srivastava. Heat-shock treatment-mediated increase in transduction by adeno-associated virus 2-vectors is independent of the cellular heat-shock protein 90. J. Biol. Chem., 279: 12714-12723, 2004.
  10. W. Zhao, L. Zhong, J. Wu, L. Chen, K. Qing, K.A. Weigel-Kelley, S.H. Larsen, W. Shou, K.H. Warrington, Jr., and A. Srivastava. Role of cellular FKBP52 protein in intracellular trafficking of recombinant adeno-associated virus 2 vectors. Virology, 353: 283-293, 2006.
  11. W. Zhao, J. Wu, L. Zhong, and A. Srivastava. Adeno-associated virus 2-mediated gene transfer: Identification of a cellular serine/threonine protein phosphatase involved in augmenting vector transduction efficiency. Gene Therapy, 14: 545-550, 2007.
  12. L. Zhong, W. Zhao, J. Wu, B. Li, B. Li, S. Zolotukhin, L. Govindasamy, M. Agbandge-McKenna, and A. Srivastava. A dual role of EGFR protein tyrosine kinase signaling in ubiquitination of AAV2 capsids and viral second-strand DNA synthesis. Mol. Therapy, 15: 1323-1330, 2007.
  13. L. Zhong, X. Zhou, Y. Li, K.Y. Qing, X. Xiao, R.J. Samulski, and A. Srivastava. Single-polarity recombinant adeno-associated virus 2 vector-mediated transgene expression in vitro and in vivo: Mechanism of transduction. Mol. Therapy, 16: 290-295, 2008.
  14. K.Y. Qing, C. Mah, J. Hansen, S.Z. Zhou, V.J. Dwarki and A. Srivastava. Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2. Nature Medicine, 5: 71-77, 1999.
  15. L. Zhong, W. Li, Z, Yang, L.Y. Chen, Y. Li, K.Y. Qing, K.A. Weigel-Kelley, M.C. Yoder, W. Shou and A. Srivastava. Improved transduction of primary murine hepatocytes by recombinant adeno-associated virus 2 vectors in vivo. Gene Therapy, 11: 1165-1169, 2004.
  16. L. Zhong, K.Y. Qing, Y. Si, L. Chen, M.Q. Tan and A. Srivastava. Heat-shock treatment-mediated increase in transduction by adeno-associated virus 2-vectors is independent of the cellular heat-shock protein 90. J. Biol. Chem., 279: 12714-12723, 2004.
  17. C. Ling, Y. Lu, J. Kalsi, G.R. Jayandharan, B. Li, W. Ma, B. Cheng, S. Gee, K. McGoogan, L. Zhong, L. Govindasamy, M. Agbandje-McKenna, and A. Srivastava. Hepatocyte growth factor receptor is a cellular coreceptor for adeno-associated virus 3. Hum. Gene Therapy, 21: 1741-1747, 2010.
  18. C. Ling, Y. Wang, Y. Lu, L. Wang, G.R. Jayandharan, G.V. Aslanidi, B. Li, B. Cheng, W. Ma, T. Lentz, C. Ling, X. Xiao, R.J. Samulski, N. Muzyczka, and A. Srivastava. The adeno-associated virus genome packaging puzzle. J. Mol. Genet. Medicine, 9: 3, 1000178, 2015.
  19. C. Ling, Z. Yin, J. Li, D. Zhang, G. Aslanidi, and A. Srivastava. Strategies to generate high-titer, high-potency recombinant AAV3 serotype vectors. Mol. Ther. Methods & Clin. Dev., 3:16029, 2016.
  20. C. Ling, Z. Yin, J. Li, D. Zhang, G. Aslanidi, and A. Srivastava. Strategies to generate high-titer, high-potency recombinant AAV3 serotype vectors. Mol. Ther. Meth. & Clin. Dev., 3:16029, 2016.
  21. H.-J. Kwon, K. Qing, S. Ponnazhagan, X.-S. Wang, D.M. Markusic, S. Gupte, S.E. Boye, and A. Srivastava. Adeno-associated virus D-sequence-mediated suppression of expression of a human major histocompatibility class II gene: Implications in the development of adeno-associated virus vectors for modulating humoral immune response. Hum. Gene Therapy, 31: 565-574, 2020.
  22. M.G. Sanal and A. Srivastava. Gene therapy for hemophilia A. New Engl. Jour. of Medicine, 386: 2247, 2022.
  23. A. Srivastava. Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy. Mol. Ther.-Nucl. Acids. 32: 949-959, 2023.
  24. A. Srivastava. AAV vectors for long-term gene therapy of hemophilia B: Are we there yet? Mol. Therapy, 32: 2042-2044, 2024.