Transduction of brain dopamine neurons by adenoviral vectors is modulated by car expression: Rationale for tropism modified vectors in PD gene therapy

PLoS ONE

Volumn 5, Issue 9, 2010, Pages 1-9

  Lewis, Travis B ^a,b,c   Glasgow, Joel N ^b,c,d,e   Glandon, Anya M ^c   Curiel, David T ^b,d   Standaert, David G ^c  

^a UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^b UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^c UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^d UNIVERSITY OF ALABAMA SCHOOL OF MEDICINE   (United States)

^e University of Alabama at Birmingham   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; COXSACKIE VIRUS AND ADENOVIRUS RECEPTOR; COXSACKIE-ADENOVIRUS RECEPTOR; DOPAMINE; VIRUS RECEPTOR;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; ASTROCYTE; CONTROLLED STUDY; DOPAMINERGIC NERVE CELL; GENE EXPRESSION; MESENCEPHALON; MOUSE; NERVE ENDING; NONHUMAN; PARKINSON DISEASE; PROTEIN EXPRESSION; PROTEIN FUNCTION; PROTEIN LOCALIZATION; SIGNAL TRANSDUCTION; SUBSTANTIA NIGRA; SYNAPTIC TRANSMISSION; TRANSGENE; TRANSGENIC MOUSE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS CELL INTERACTION; WILD TYPE; ADENOVIRUS; ANIMAL; BRAIN; C57BL MOUSE; DISEASE MODEL; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HUMAN; METABOLISM; NERVE CELL; PHYSIOLOGY; PROTEIN BINDING; VIRAL TROPISM; VIROLOGY;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; ANIMALIA;

ADENOVIRIDAE; ANIMALS; BRAIN; DISEASE MODELS, ANIMAL; DOPAMINE; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; HUMANS; MICE; MICE, INBRED C57BL; MICE, TRANSGENIC; NEURONS; PARKINSON DISEASE; PROTEIN BINDING; RECEPTORS, VIRUS; TRANSDUCTION, GENETIC; VIRAL TROPISM;

EID: 77958606862     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0012672     Document Type: Article

References (47)

1. Lewis TB, Standaert DG (2008) Design of clinical trials of gene therapy in Parkinson disease. Exp Neurol 209: 41-47.
2. Bjorklund T, Kordower JH (2010) Gene therapy for Parkinson's disease. Mov Disord 25 Suppl 1: S161-173.
3. Christine CW, Starr PA, Larson PS, Eberling JL, Jagust WJ, et al. (2009) Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology 73: 1662-1669.
4. Eberling JL, Jagust WJ, Christine CW, Starr P, Larson P, et al. (2008) Results from a phase I safety trial of hAADC gene therapy for Parkinson disease. Neurology 70: 1980-1983.
5. Kaplitt MG, Feigin A, Tang C, Fitzsimons HL, Mattis P, et al. (2007) Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369: 2097-2105.
6. Oxford BioMedica (2010) Phase I/II Study of the Safety, Efficacy and Dose Evaluation of ProSavin for the Treatment of Bilateral Idiopathic Parkinson's Disease. Available: http://clinicaltrials.gov/ct2/show/NCT00627588. Accessed 2010 August 20.
7. Neurologix, Inc (2010) Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease. Available: http://clinicaltrials.gov/ct2/show/NCT00643890. Accessed 2010 August 20.
8. Genzyme (2010) A Study of AAV-hAADC-2 in Subjects With Parkinson's Disease. Available: http://clinicaltrials.gov/ct2/show/NCT00229736. Accessed 2010 August 20.
9. Neurologix, Inc., Weill Medical College of Cornell University North Shore University Hospital (2010) Safety Study of Subthalamic Nucleus Gene Therapy for Parkinson's Disease. Available: http://clinicaltrials.gov/ct2/show/NCT00195143. Accessed 2010 August 20.
10. Marks WJ Jr., Ostrem JL, Verhagen L, Starr PA, Larson PS, et al. (2008) Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol 7: 400-408.
11. Ceregene (2010) Double-Blind, Multicenter, Sham Surgery Controlled Study of CERE-120 For Efficacy and Safety in Subjects With Idiopathic Parkinson's Disease. Available: http://clinicaltrials.gov/ct2/show/NCT00400634. Accessed 2010 August 20.
12. Ceregene (2010) Safety of CERE-120 (AAV2-NTN) in Subjects With Idiopathic Parkinson's Disease. Available: http://clinicaltrials.gov/ct2/show/NCT00252850. Accessed 2010 August 20.
13. Ceregene (2010) Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease. Available: http://clinicaltrials.gov/ct2/show/NCT00985517. Accessed 2010 August 20.
14. Reynolds PN, Nicklin SA, Kaliberova L, Boatman BG, Grizzle WE, et al. (2001) Combined transductional and transcriptional targeting improves the specificity of transgene expression in vivo. Nat Biotechnol 19: 838-842.
15. Glasgow JN, Everts M, Curiel DT (2006) Transductional targeting of adenovirus vectors for gene therapy. Cancer Gene Ther 13: 830-844.
16. Akli S, Caillaud C, Vigne E, Stratford-Perricaudet LD, Poenaru L, et al. (1993) Transfer of a foreign gene into the brain using adenovirus vectors. Nat Genet 3: 224-228.
17. Soudais C, Laplace-Builhe C, Kissa K, Kremer EJ (2001) Preferential transduction of neurons by canine adenovirus vectors and their efficient retrograde transport in vivo. FASEB J 15: 2283-2285.
18. Smith-Arica JR, Morelli AE, Larregina AT, Smith J, Lowenstein PR, et al. (2000) Cell-type-specific and regulatable transgenesis in the adult brain: adenovirus-encoded combined transcriptional targeting and inducible transgene expression. Mol Ther 2: 579-587.
19. Tomko RP, Xu R, Philipson L (1997) HCAR and MCAR: the human and mouse cellular receptors for subgroup C adenoviruses and group B coxsackieviruses. Proc Natl Acad Sci U S A 94: 3352-3356.
20. Bergelson JM, Cunningham JA, Droguett G, Kurt-Jones EA, Krithivas A, et al. (1997) Isolation of a common receptor for Coxsackie B viruses and adenoviruses 2 and 5. Science 275: 1320-1323.
21. Paul CP, Everts M, Glasgow JN, Dent P, Fisher PB, et al. (2008) Characterization of infectivity of knob-modified adenoviral vectors in glioma. Cancer Biol Ther 7: 786-793.
22. Wu H, Seki T, Dmitriev I, Uil T, Kashentseva E, et al. (2002) Double modification of adenovirus fiber with RGD and polylysine motifs improves coxsackievirus-adenovirus receptor-independent gene transfer efficiency. Hum Gene Ther 13: 1647-1653.
23. Li HJ, Everts M, Pereboeva L, Komarova S, Idan A, et al. (2007) Adenovirus tumor targeting and hepatic untargeting by a coxsackie/adenovirus receptor ectodomain anti-carcinoembryonic antigen bispecific adapter. Cancer Res 67: 5354-5361.
24. Hedley SJ, Auf der Maur A, Hohn S, Escher D, Barberis A, et al. (2006) An adenovirus vector with a chimeric fiber incorporating stabilized single chain antibody achieves targeted gene delivery. Gene Ther 13: 88-94.
25. Glasgow JN, Kremer EJ, Hemminki A, Siegal GP, Douglas JT, et al. (2004) An adenovirus vector with a chimeric fiber derived from canine adenovirus type 2 displays novel tropism. Virology 324: 103-116.
26. Hotta Y, Honda T, Naito M, Kuwano R (2003) Developmental distribution of coxsackie virus and adenovirus receptor localized in the nervous system. Brain Res Dev Brain Res 143: 1-13.
27. Tallone T, Malin S, Samuelsson A, Wilbertz J, Miyahara M, et al. (2001) A mouse model for adenovirus gene delivery. Proc Natl Acad Sci U S A 98: 7910-7915.
28. Graybiel AM, Hirsch EC, Agid Y (1990) The nigrostriatal system in Parkinson's disease. Adv Neurol 53: 17-29.
29. Jimenez-Castellanos J, Graybiel AM (1987) Subdivisions of the dopamine-containing A8-A9-A10 complex identified by their differential mesostriatal innervation of striosomes and extrastriosomal matrix. Neuroscience 23: 223-242.
30. Hammond R, Blaess S, Abeliovich A (2009) Sonic hedgehog is a chemoat-tractant for midbrain dopaminergic axons. PLoS One 4: e7007.
31. Honda T, Saitoh H, Masuko M, Katagiri-Abe T, Tominaga K, et al. (2000) The coxsackievirus-adenovirus receptor protein as a cell adhesion molecule in the developing mouse brain. Brain Res Mol Brain Res 77: 19-28
32. Dmitriev I, Krasnykh V, Miller CR, Wang M, Kashentseva E, et al. (1998) An adenovirus vector with genetically modified fibers demonstrates expanded tropism via utilization of a coxsackievirus and adenovirus receptor-independent cell entry mechanism. J Virol 72: 9706-9713.
33. Cripe TP, Dunphy EJ, Holub AD, Saini A, Vasi NH, et al. (2001) Fiber knob modifications overcome low, heterogeneous expression of the coxsackievirus-adenovirus receptor that limits adenovirus gene transfer and oncolysis for human rhabdomyosarcoma cells. Cancer Res 61: 2953-2960.
34. Guse K, Diaconu I, Rajecki M, Sloniecka M, Hakkarainen T, et al. (2009) Ad5/ 3-9HIF-Delta24-VEGFR-1-Ig, an infectivity enhanced, dual-targeted and antiangiogenic oncolytic adenovirus for kidney cancer treatment. Gene Ther 16: 1009-1020.
35. Kanerva A, Lavilla-Alonso S, Raki M, Kangasniemi L, Bauerschmitz GJ, et al. (2008) Systemic therapy for cervical cancer with potentially regulatable oncolytic adenoviruses. PLoS One 3: e2917.
36. Ulasov IV, Rivera AA, Han Y, Curiel DT, Zhu ZB, et al. (2007) Targeting adenovirus to CD80 and CD86 receptors increases gene transfer efficiency to malignant glioma cells. J Neurosurg 107: 617-627.
37. Stoff-Khalili MA, Rivera AA, Glasgow JN, Le LP, Stoff A, et al. (2005) A human adenoviral vector with a chimeric fiber from canine adenovirus type 1 results in novel expanded tropism for cancer gene therapy. Gene Ther 12: 1696-1706.
38. Pesonen S, Nokisalmi P, Escutenaire S, Sarkioja M, Raki M, et al. Prolonged systemic circulation of chimeric oncolytic adenovirus Ad5/3-Cox2L-D24 in patients with metastatic and refractory solid tumors. Gene Ther 17: 892-904.
39. University of Alabama at Birmingham, National Cancer Institute (2010) A Study of an Infectivity Enhanced Suicide Gene Expressing Adenovirus for Ovarian Cancer in Patients With Recurrent Ovarian and Other Selected Gynecologic Cancers. Available: http://clinicaltrials.gov/ct2/show/NCT00964756. Accessed 2010 August 20.
40. Fields BN, Knipe DM, Howley PM (1996) Fields virology. Philadelphia: Lippincott-Raven Publishers, 2 v. p.1146.
41. Su X, Kells AP, Huang EJ, Lee HS, Hadaczek P, et al. (2009) Safety evaluation of AAV2-GDNF gene transfer into the dopaminergic nigrostriatal pathway in aged and parkinsonian rhesus monkeys. Hum Gene Ther 20: 1627-1640.
42. Manfredsson FP, Tumer N, Erdos B, Landa T, Broxson CS, et al. (2009) Nigrostriatal rAAV-mediated GDNF overexpression induces robust weight loss in a rat model of age-related obesity. Mol Ther 17: 980-991.
43. American Society of Gene Therapy (2009) Abstracts of the American Society of Gene Therapy 12th Annual Meeting. May 27-30, 2009. San Diego, California, USA. Mol Ther 17 Suppl 1: S1-396.
44. Slevin JT, Gash DM, Smith CD, Gerhardt GA, Kryscio R, et al. (2007) Unilateral intraputamenal glial cell line-derived neurotrophic factor in patients with Parkinson disease: response to 1 year of treatment and 1 year of withdrawal. J Neurosurg 106: 614-620.
45. Nutt JG, Burchiel KJ, Comella CL, Jankovic J, Lang AE, et al. (2003) Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. Neurology 60: 69-73.
46. Tatarewicz SM, Wei X, Gupta S, Masterman D, Swanson SJ, et al. (2007) Development of a maturing T-cell-mediated immune response in patients with idiopathic Parkinson's disease receiving r-metHuGDNF via continuous intra-putaminal infusion. J Clin Immunol 27: 620-627.
47. Kordower JH (2003) In vivo gene delivery of glial cell line-derived neurotrophic factor for Parkinson's disease. Ann Neurol 53 Suppl 3: S120-132; discussion S132-124.