Single point mutation in adeno-associated viral vectors -DJ capsid leads to improvement for gene delivery in vivo

BMC Biotechnology

Volumn 16, Issue 1, 2016, Pages

  Mao, Yingying ^a   Wang, Xuejun ^b   Yan, Renhe ^a   Hu, Wei ^b   Li, Andrew ^c   Wang, Shengqi ^b   Li, Hongwei ^a  

^a SOUTHERN MEDICAL UNIVERSITY   (China)

^b BEIJING INSTITUTE OF RADIATION MEDICINE   (China)

^c JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Adeno associated virus DJ; Gene delivery; Improvement; In vivo; Point mutation

Indexed keywords

BACTERIOPHAGES; BIOLUMINESCENCE; EFFICIENCY; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; MAMMALS;

ADENO-ASSOCIATED VIRUS; GENE DELIVERY; IMPROVEMENT; IN-VIVO; POINT MUTATIONS;

VECTORS;

FIREFLY LUCIFERASE; GAUSSIA LUCIFERASE; GREEN FLUORESCENT PROTEIN; LUCIFERASE; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG; CAPSID PROTEIN;

ADENO ASSOCIATED VIRAL VECTOR DJ CAPSID; ANIMAL EXPERIMENT; ARTICLE; BIOLUMINESCENCE; CONTROLLED STUDY; GENE DOSAGE; GENETIC TRANSDUCTION; HEK293 CELL LINE; HELA CELL LINE; HEPG2 CELL LINE; HUMAN; HUMAN CELL; IMAGE ANALYSIS; IN VITRO GENE TRANSFER; IN VIVO GENE TRANSFER; MALE; MOUSE; MUTANT; NONHUMAN; POINT MUTATION; PROTEIN EXPRESSION; VIRAL TROPISM; VIRUS CAPSID; VIRUS GENOME; ANIMAL; C57BL MOUSE; CELL LINE; DEPENDOPARVOVIRUS; FLUORESCENCE IMAGING; GENETIC TRANSFECTION; GENETICS; HEP-G2 CELL LINE; METABOLISM; PROCEDURES; TISSUE DISTRIBUTION;

ANIMALS; CAPSID PROTEINS; CELL LINE; DEPENDOVIRUS; GENOME, VIRAL; HELA CELLS; HEP G2 CELLS; HUMANS; LUCIFERASES; MALE; MICE; MICE, INBRED C57BL; OPTICAL IMAGING; POINT MUTATION; TISSUE DISTRIBUTION; TRANSFECTION;

EID: 84959152780     PISSN: None     EISSN: 14726750     Source Type: Journal    
DOI: 10.1186/s12896-015-0230-0     Document Type: Article

References (34)

1. Sobrevals L, Enguita M, Rodriguez C, Gonzalez-Rojas J, Alzaguren P, Razquin N, et al. AAV vectors transduce hepatocytes in vivo as efficiently in cirrhotic as in healthy rat livers. Gene Ther. 2012;19(4):411-7.
2. Wang Z, Zhu T, Qiao C, Zhou L, Wang B, Zhang J, et al. Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat Biotechnol. 2005;23(3):321-8.
3. Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, et al. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther. 2010;18(3):643-50.
4. Fang H, Lai NC, Gao MH, Miyanohara A, Roth DM, Tang T, et al. Comparison of adeno-associated virus serotypes and delivery methods for cardiac gene transfer. Human Gene Ther Methods. 2012;23(4):234-41.
5. Picconi JL, Muff-Luett MA, Wu D, Bunchman E, Schaefer F, Brophy PD. Kidney-specific expression of GFP by in-utero delivery of pseudotyped adeno-associated virus 9. Mol Ther Methods Clin Dev. 2014;1:14014.
6. Lipkowitz MS, Hanss B, Tulchin N, Wilson PD, Langer JC, Ross MD, et al. Transduction of renal cells in vitro and in vivo by adeno-associated virus gene therapy vectors. J Am Soc Nephrol. 1999;10(9):1908-15.
7. Wu Z, Asokan A, Samulski RJ. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther. 2006;14(3):316-27.
8. Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med. 2008;358(21):2231-9.
9. Maguire AM, Simonelli F, Pierce EA, Pugh Jr EN, Mingozzi F, Bennicelli J, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med. 2008;358(21):2240-8.
10. Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 2011;365(25):2357-65.
11. Daya S, Berns KI. Gene therapy using adeno-associated virus vectors. Clin Microbiol Rev. 2008;21(4):583-93.
12. Zhong L, Li B, Mah CS, Govindasamy L, Agbandje-McKenna M, Cooper M, et al. Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci U S A. 2008;105(22):7827-32.
13. Markusic DM, Herzog RW, Aslanidi GV, Hoffman BE, Li B, Li M, et al. High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines. Mol Ther. 2010;18(12):2048-56.
14. Gabriel N, Hareendran S, Sen D, Gadkari RA, Sudha G, Selot R, et al. Bioengineering of AAV2 capsid at specific serine, threonine, or lysine residues improves its transduction efficiency in vitro and in vivo. Hum Gene Ther Methods. 2013;24(2):80-93.
15. Sen D, Gadkari RA, Sudha G, Gabriel N, Kumar YS, Selot R, et al. Targeted modifications in adeno-associated virus serotype 8 capsid improves its hepatic gene transfer efficiency in vivo. Hum Gene Ther Methods. 2013;24(2):104-16.
16. Sen D, Balakrishnan B, Gabriel N, Agrawal P, Roshini V, Samuel R, et al. Improved adeno-associated virus (AAV) serotype 1 and 5 vectors for gene therapy. Sci Rep. 2013;3:1832.
17. Wu Z, Asokan A, Grieger JC, Govindasamy L, Agbandje-McKenna M, Samulski RJ. Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J Virol. 2006;80(22):11393-7.
18. Aslanidi GV, Rivers AE, Ortiz L, Govindasamy L, Ling C, Jayandharan GR, et al. High-efficiency Transduction of Human Monocyte-derived Dendritic Cells by Capsid-modified Recombinant AAV2 Vectors. 2012.
19. Grimm D, Lee JS, Wang L, Desai T, Akache B, Storm TA, et al. In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J Virol. 2008;82(12):5887-911.
20. Hickey RD, Lillegard JB, Fisher JE, McKenzie TJ, Hofherr SE, Finegold MJ, et al. Efficient production of Fah-null heterozygote pigs by chimeric adeno-associated virus-mediated gene knockout and somatic cell nuclear transfer. Hepatology. 2011;54(4):1351-9.
21. Melo SP, Lisowski L, Bashkirova E, Zhen HH, Chu K, Keene DR, et al. Somatic correction of junctional epidermolysis bullosa by a highly recombinogenic AAV variant. Mol Ther. 2014;22(4):725-33.
22. Ayuso E, Mingozzi F, Bosch F. Production, Purification and Characterization of Adeno-Associated Vectors. Current Gene Ther. 2010;10(6):423-36.
23. Dickey DD, Excoffon KJ, Young KR, Parekh KR, Zabner J. Hoechst increases adeno-associated virus-mediated transgene expression in airway epithelia by inducing the cytomegalovirus promoter. J Gene Med. 2012;14(6):366-73.
24. Alexander IE, Russell DW, Miller AD. DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors. J Virol. 1994;68(12):8282-7.
25. Moulay G, Boutin S, Masurier C, Scherman D, Kichler A. Polymers for improving the in vivo transduction efficiency of AAV2 vectors. PLoS ONE. 2010;5(12):e15576.
26. Zhong L, Jayandharan GR, Aslanidi GV, Zolotukhin S, Herzog RW, Srivastava A. Development of Novel Recombinant AAV Vectors and Strategies for the Potential Gene Therapy of Hemophilia. J Genet Syndr Gene Ther. 2012;S1.
27. Ahmadiankia NNV, Neshati Z, Swildens J, de Vries AA. Generation of Helper Plasmids Encoding Mutant Adeno-associated Virus Type 2 Capsid Proteins with Increased Resistance against Proteasomal Degradation. Iran J Basic Med Sci. 2013;16(7):813-21.
28. Aslanidi GV, Rivers AE, Ortiz L, Govindasamy L, Ling C, Jayandharan GR, et al. High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors. Vaccine. 2012;30(26):3908-17.
29. Petrs-Silva H, Dinculescu A, Li Q, Min SH, Chiodo V, Pang JJ, et al. High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther. 2009;17(3):463-71.
30. Aslanidi GV, Rivers AE, Ortiz L, Song L, Ling C, Govindasamy L, et al. Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: the final threshold? PLoS ONE. 2013;8(3):e59142.
31. Lerch TF, O'Donnell JK, Meyer NL, Xie Q, Taylor KA, Stagg SM, et al. Structure of AAV-DJ, a retargeted gene therapy vector: cryo-electron microscopy at 4.5 A resolution. Structure. 2012;20(8):1310-20.
32. Kauss MA, Smith LJ, Zhong L, Srivastava A, Wong Jr KK, Chatterjee S. Enhanced long-term transduction and multilineage engraftment of human hematopoietic stem cells transduced with tyrosine-modified recombinant adeno-associated virus serotype 2. Hum Gene Ther. 2010;21(9):1129-36.
33. Qiao C, Yuan Z, Li J, Tang R, Li J, Xiao X. Single tyrosine mutation in AAV8 and AAV9 capsids is insufficient to enhance gene delivery to skeletal muscle and heart. Human Gene Ther Methods. 2012;23(1):29-37.
34. Qiao C, Li C, Zhao C, Li J, Bian T, Grieger J, et al. K137R mutation on adeno-associated viral capsids had minimal effect on enhancing gene delivery in vivo. Human Gene Ther Methods. 2014;25(1):33-9.