Advancements in adeno-associated viral gene therapy approaches: Exploring a new horizon

F1000 Medicine Reports

Volumn 3, Issue 1, 2011, Pages

  Aalbers, Caroline J ^a,b   Tak, Paul P ^a,b   Vervoordeldonk, Margriet J ^a,b  

^a UNIVERSITY OF AMSTERDAM   (Netherlands)

^b Arthrogen BV   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

ALIPOGENE TIPARVOVEC; CYCLOSPORIN; METHYLPREDNISOLONE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; PARVOVIRUS VECTOR;

ALPHA 1 ANTITRYPSIN DEFICIENCY; ALZHEIMER DISEASE; CANAVAN DISEASE; CELL TRANSFER; CLINICAL TRIAL (TOPIC); CYSTIC FIBROSIS; DRUG DOSE ESCALATION; GENE TRANSFER; HEMOPHILIA B; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTED PATIENT; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; HYPERLIPOPROTEINEMIA TYPE 1; LEBER CONGENITAL AMAUROSIS; MELANOMA; MUSCULAR DYSTROPHY; NEURONAL CEROID LIPOFUSCINOSIS; NONHUMAN; PARKINSON DISEASE; REVIEW; RHEUMATOID ARTHRITIS; VIRAL GENE THERAPY;

EID: 80053492356     PISSN: None     EISSN: 17575931     Source Type: Journal    
DOI: 10.3410/M3-17     Document Type: Review

References (33)

1. Adriaansen J, Vervoordeldonk MJ, Tak PP: Gene therapy as therapeutic approach for the treatment of rheumatoid arthritis: innovative vectors and therapeutic genes. Rheumatology (Oxford) 2006, 45:656-68. (Pubitemid 43985333)
2. Gene Therapy Clinical Trials Worldwide -The Journal of Gene Medicine Clinical Trial site. [www.abedia.com/wiley/index. html]
3. Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JI, Hauck B, Zelenaia O, Zhu X, Raffini L, Coppieters F, De Baere E, Shindler KS, Volpe NJ, Surace EM, Acerra C, Lyubarsky A, Redmond TM, Stone E, Sun J, McDonnell JW, et al.: Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 2009, 374:1597-605. F1000 Factor 8 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
4. Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, Viswanathan A, Holder GE, Stockman A, Tyler N, Petersen-Jones S, Bhattacharya SS, Thrasher AJ, Fitzke FW, Carter BJ, Rubin GS, Moore AT, Ali RR: Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 2008, 358:2231-9. F1000 Factor 8 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
5. Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, Rossi S, Marshall K, Banfi S, Surace EM, Sun J, Redmond TM, Zhu X, Shindler KS, Ying GS, Ziviello C, Acerra C, Wright JF, McDonnell JW, High KA, Bennett J, Auricchio A: Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther 2010, 18:643-50. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
6. Stieger K, Lorenz B: Gene therapy for vision loss - recent developments. Discov Med 2010, 10:425-33. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
7. Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DG, Johnson FA, McClelland A, Scallan C, Skarsgard E, Flake AW, Kay MA, High KA, Glader B: AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 2003, 101:2963-72. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
8. Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, 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. F1000 Factor Evaluated by Margriet Vervoordeldonk 19 Aug 2011
9. Mingozzi F, Maus MV, Hui DJ, Sabatino DE, Murphy SL, Rasko JE, Ragni MV, Manno CS, Sommer J, Jiang H, Pierce GF, Ertl HC, High KA: CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med 2007, 13:419-22. F1000 Factor 8 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
10. Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Mingozzi F, Ng CY, Zhou J, Spence Y, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, Davidoff AM: Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins. Mol Ther 2011, 19:876-85. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
11. Allay JA, Sleep S, Long S, Tillman DM, Clark R, Carney G, Fagone P, McIntosh JH, Nienhuis AW, Davidoff AM, Nathwani AC, Gray JT: Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial. Hum Gene Ther 2011, 22:595-604.
12. Stroes ES, Nierman MC, Meulenberg JJ, Franssen R, Twisk J, Henny CP, Maas MM, Zwinderman AH, Ross C, Aronica E, High KA, Levi MM, Hayden MR, Kastelein JJ, Kuivenhoven JA: Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients. Arterioscler Thromb Vasc Biol 2008, 28:2303-4. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
13. Mingozzi F, Meulenberg JJ, Hui DJ, Basner-Tschakarjan E, Hasbrouck NC, Edmonson SA, Hutnick NA, Betts MR, Kastelein JJ, Stroes ES, High KA: AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells. Blood 2009, 114:2077-86. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
14. Gaudet D, de Wal J, Tremblay K, Déry S, van Deventer S, Freidig A, Brisson D, Méthot J: Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency. Atheroscler Suppl 2010, 11:55-60. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
15. Chan JMK, Villareal G, Jin WW, Stepan T, Burstein H, Wahl SM: Intraarticular gene transfer of TNFR:Fc suppresses experimental arthritis with reduced systemic distribution of the gene product. Mol Ther 2002, 6:727-36. (Pubitemid 36054306)
16. Adriaansen J, Tas SW, Klarenbeek PL, Bakker AC, Apparailly F, Firestein GS, Jorgensen C, Vervoordeldonk MJ, Tak PP: Enhanced gene transfer to arthritic joints using adeno-associated virus type 5: implications for intra-articular gene therapy. Ann Rheum Dis 2005, 64:1677-84. (Pubitemid 41667139)
17. Apparailly F, Khoury M, Vervoordeldonk MJ, Adriaansen J, Gicquel E, Perez N, Riviere C, Louis-Plence P, Noel D, Danos O, Douar AM, Tak PP, Jorgensen C: Adeno-associated virus pseudotype 5 vector improves gene transfer in arthritic joints. Hum Gene Ther 2005, 16:426-34. (Pubitemid 40647140)
18. Goodrich LR, Choi VW, Carbone BA, McIlwraith CW, Samulski RJ: Ex vivo serotype-specific transduction of equine joint tissue by self-complementary adeno-associated viral vectors. Hum Gene Ther 2009, 20:1697-702. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
19. Boutin S, Monteilhet V, Veron P, Leborgne C, Benveniste O, Montus MF, Masurier C: 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. F1000 Factor 8 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
20. Jiang H, Couto LB, Patarroyo-White S, Liu T, Nagy D, Vargas JA, Zhou S, Scallan CD, Sommer J, Vijay S, Mingozzi F, High KA, Pierce GF: Effects of transient immunosuppression on AAV-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. Blood 2006, 108:3321-8. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
21. Adriaansen J, Khoury M, De Cortie CJ, Fallaux FJ, Bigey P, Apparailly F, Jorgensen C, Vervoordeldonk MJ, Tak PP: Reduction of arthritis following intra-articular administration of an adeno-associated virus serotype 5 expressing a disease-inducible TNF-blocking agent. Ann Rheum Dis 2007, 66:1143-50. (Pubitemid 47309728)
22. Khoury M, Adriaansen J, Vervoordeldonk MJ, Gould D, Chernajovsky Y, Bigey P, Bloquel C, Sherman D, Tak PP, Jorgenson C, Apparailly F: Inflammation-inducible anti-TNF gene expression mediated by intra-articular injection of serotype 5 AAV reduces arthritis. J Gene Med 2007, 9:596-604. (Pubitemid 47073992)
23. Adriaansen J, Fallaux FJ, De Cortie CJ, Vervoordeldonk MJ, Tak PP: Local delivery of beta interferon using an adeno-associated virus type 5 effectively inhibits adjuvant arthritis in rats. J Gen Virol 2007, 88:1717-21. (Pubitemid 46845975)
24. Evans CH, Robbins PD, Ghivizzani SC, Wasko MC, Tomaino MM, Kang R, Muzzonigro TA, Vogt M, Elder EM, Whiteside TL, Watkins SC, Herndon JH: Gene transfer to human joints: progress toward a gene therapy of arthritis. Proc Natl Acad Sci U S A 2005, 102:8698-703. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
25. Mease PJ, Hobbs K, Chalmers A, El-Gabalawy H, Bookman A, Keystone E, Furst DE, Anklesaria P, Heald AE: Local delivery of a recombinant adenoassociated vector containing a tumour necrosis factor alpha antagonist gene in inflammatory arthritis: a phase 1 dose-escalation safety and tolerability study. Ann Rheum Dis 2009, 68:1247-54. F1000 Factor 9 Evaluated by Ulf Mueller-Ladner 05 Aug 2009, Margriet Vervoordeldonk 19 Aug 2011
26. Mease PJ, Wei N, Fudman EJ, Kivitz AJ, Schechtman J, Trapp RG, Hobbs KF, Greenwald M, Hou A, Bookbinder SA, Graham GE, Wiesenhutter CW, Willis L, Ruderman EM, Forstot JZ, Maricic MJ, Dao KH, Pritchard CH, Fiske DN, Burch FX, Prupas HM, Anklesaria P, Heald AE: Safety, tolerability, and clinical outcomes after intraarticular injection of a recombinant adeno-associated vector containing a tumor necrosis factor antagonist gene: results of a phase 1/2 Study. J Rheumatol 2010, 37:692-703. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
27. Frank KM, Hogarth DK, Miller JL, Mandal S, Mease PJ, Samulski RJ, Weisgerber GA, Hart J: Investigation of the cause of death in a gene-therapy trial. N Engl J Med 2009, 361:161-9. F1000 Factor Evaluated by Margriet Vervoordeldonk 19 Aug 2011
28. DiPrimio N, McPhee SW, Samulski RJ: Adeno-associated virus for the treatment of muscle diseases: toward clinical trials. Curr Opin Mol Ther 2010, 12:553-60. F1000 Factor 8 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
29. Märsch S, Huber A, Büning H, Hallek M, Perabo L: Optimization of stealth adeno-associated virus vectors by randomization of immunogenic epitopes. Virology 2010, 397:167-75. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
30. LoDuca PA, Hoffman BE, Herzog RW: Hepatic gene transfer as a means of tolerance induction to transgene products. Curr Gene Ther 2009, 9:104-14. F1000 Factor 8 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
31. Märsch S, Huber A, Hallek M, Büning H, Perabo L: A novel directed evolution method to enhance cell-type specificity of adeno-associated virus vectors. Comb Chem High Throughput Screen 2010, 13:807-12. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
32. Deichmann A, Hacein-Bey-Abina S, Schmidt M, Garrigue A, Brugman MH, Hu J, Glimm H, Gyapay G, Prum B, Fraser CC, Fischer N, Schwarzwaelder K, Siegler ML, De Ridder D, Pike- Overzet K, Howe SJ, Thrasher AJ, Wagemaker G, Abel U, Staal FJ, Delabesse E, Villeval JL, Aronow B, Hue C, Prinz C, Wissler M, Klanke C, Weissenbach J, Alexander I, Fischer A, et al.: Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J Clin Invest 2007, 117:2225-32. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011
33. Li H, Malani N, Hamilton SR, Schlachterman A, Bussadori G, Edmonson SE, Shah R, Arruda VR, Mingozzi F, Wright JF, Bushman FD, High KA: Assessing the potential for AAV vector genotoxicity in a murine model. Blood 2011, 117:3311-9. F1000 Factor 6 Evaluated by Margriet Vervoordeldonk 19 Aug 2011