Real-time MR imaging of adeno-associated viral vector delivery to the primate brain

NeuroImage

Volumn 47, Issue SUPPL. 2, 2009, Pages

  Fiandaca, Massimo S ^a   Varenika, Vanja ^a   Eberling, Jamie ^a,b   McKnight, Tracy ^a   Bringas, John ^a   Pivirotto, Phillip ^a   Beyer, Janine ^a   Hadaczek, Piotr ^a   Bowers, William ^c   Park, John ^d   Federoff, Howard ^e   Forsayeth, John ^a   Bankiewicz, Krystof S ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b LAWRENCE BERKELEY NATIONAL LABORATORY   (United States)

^c UNIVERSITY OF ROCHESTER   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e GEORGETOWN UNIVERSITY   (United States)

Author keywords

Adeno associated virus; Corona radiata; Gadolinium; Liposomes; Putamen; Thalamus

Indexed keywords

ADENOVIRUS VECTOR; AMINO ACID DECARBOXYLASE; GADOLINIUM; GREEN FLUORESCENT PROTEIN; LIPOSOME;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; IMMUNOHISTOCHEMISTRY; IN VIVO STUDY; MALE; NEUROIMAGING; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; POSITRON EMISSION TOMOGRAPHY; PRIMATE; PRIORITY JOURNAL; PUTAMEN; THALAMUS; VIRAL GENE DELIVERY SYSTEM; VIRUS PARTICLE; WHITE MATTER;

ANIMALS; AROMATIC-L-AMINO-ACID DECARBOXYLASES; BRAIN; DEPENDOVIRUS; GADOLINIUM; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HUMANS; IMMUNOHISTOCHEMISTRY; LIPOSOMES; MACACA MULATTA; MAGNETIC RESONANCE IMAGING; MALE; POSITRON-EMISSION TOMOGRAPHY; PUTAMEN; THALAMUS;

EID: 67651172965     PISSN: 10538119     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.neuroimage.2008.11.012     Document Type: Article

References (46)

1. Baekelandt V., De Strooper B., Nuttin B., and Debyser Z. Gene therapeutic strategies for neurodegenerative diseases. Curr. Opin. Mol. Ther. 2 (2000) 540-554
2. Bankiewicz K.S., Oldfield E.H., Chiueh C.C., Doppman J.L., Jacobowitz D.M., and Kopin I.J. Hemiparkinsonism in monkeys after unilateral internal carotid artery infusion of 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine (MPTP). Life Sci. 39 (1986) 7-16
3. Bankiewicz K.S., Eberling J.L., Kohutnicka M., Jagust W., Pivirotto P., Bringas J., Cunningham J., Budinger T.F., and Harvey-White J. Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach. Exp. Neurol. 164 (2000) 2-14
4. Bankiewicz K.S., Forsayeth J., Eberling J.L., Sanchez-Pernaute R., Pivirotto P., Bringas J., Herscovitch P., Carson R.E., Eckelman W., Reutter B., and Cunningham J. Long-Term Clinical Improvement in MPTP-Lesioned Primates after Gene Therapy with AAV-hAADC. Mol. Ther. 14 (2006) 564-570
5. Bobo R.H., Laske D.W., Akbasak A., Morrison P.F., Dedrick R.L., and Oldfield E.H. Convection-enhanced delivery of macromolecules in the brain. Proc. Natl. Acad. Sci. U. S. A. 91 (1994) 2076-2080
6. Carter B.J. Adeno-associated virus vectors in clinical trials. Hum. Gene. Ther. 16 (2005) 541-550
7. Chen M.Y., Lonser R.R., Morrison P.F., Governale L.S., and Oldfield E.H. Variables affecting convection-enhanced delivery to the striatum: a systematic examination of rate of infusion, cannula size, infusate concentration, and tissue-cannula sealing time. J. Neurosurg. 90 (1999) 315-320
8. Eberling J.L., Jagust W.J., Christine C.W., Starr P., Larson P., Bankiewicz K.S., and Aminoff M.J. Results from a phase I safety trial of hAADC gene therapy for Parkinson's disease. Neurology 70 (2008) 1980-1983
9. Fiandaca M., Forsayeth J., and Bankiewicz K. Current status of gene therapy trials for Parkinson's disease. Exp. Neurol. 209 (2008) 51-57
10. Forsayeth J.R., Eberling J.L., Sanftner L.M., Zhen Z., Pivirotto P., Bringas J., Cunningham J., and Bankiewicz K.S. A dose-ranging study of AAV-hAADC therapy in Parkinsonian monkeys. Mol. Ther. 14 (2006) 571-577
11. Gao G., Lu Y., Calcedo R., Grant R.L., Bell P., Wang L., Figueredo J., Lock M., and Wilson J.M. Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. Mol. Ther. 13 (2006) 77-87
12. Grieger J.C., and Samulski R.J. Adeno-associated virus as a gene therapy vector: vector development, production and clinical applications. Adv. Biochem. Eng. Biotechnol. 99 (2005) 119-145
13. Grimm D., and Kay M.A. From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Curr. Gene Ther. 3 (2003) 281-304
14. Hadaczek P., Kohutnicka M., Krauze M.T., Bringas J., Pivirotto P., Cunningham J., and Bankiewicz K. Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain. Hum. Gene Ther. 17 (2006) 291-302
15. Hadaczek P., Yamashita Y., Mirek H., Tamas L., Bohn M.C., Noble C., Park J.W., and Bankiewicz K. The "perivascular pump" driven by arterial pulsation is a powerful mechanism for the distribution of therapeutic molecules within the brain. Mol. Ther. 14 (2006) 69-78
16. Hamilton J.F., Morrison P.F., Chen M.Y., Harvey-White J., Pernaute R.S., Phillips H., Oldfield E., and Bankiewicz K.S. Heparin coinfusion during convection-enhanced delivery (CED) increases the distribution of the glial-derived neurotrophic factor (GDNF) ligand family in rat striatum and enhances the pharmacological activity of neurturin. Exp. Neurol. 168 (2001) 155-161
17. Krauze M.T., McKnight T.R., Yamashita Y., Bringas J., Noble C.O., Saito R., Geletneky K., Forsayeth J., Berger M.S., Jackson P., Park J.W., and Bankiewicz K.S. Real-time visualization and characterization of liposomal delivery into the monkey brain by magnetic resonance imaging. Brain Res. Brain Res. Protoc. 16 (2005) 20-26
18. Krauze M.T., Saito R., Noble C., Tamas M., Bringas J., Park J.W., Berger M.S., and Bankiewicz K. Reflux-free cannula for convection-enhanced high-speed delivery of therapeutic agents. J. Neurosurg. 103 (2005) 923-929
19. Krauze M.T., Forsayeth J., Park J.W., and Bankiewicz K.S. Real-time imaging and quantification of brain delivery of liposomes. Pharm. Res. 23 (2006) 2493-2504
20. Krauze M.T., Vandenberg S.R., Yamashita Y., Saito R., Forsayeth J., Noble C., Park J., and Bankiewicz K.S. Safety of real-time convection-enhanced delivery of liposomes to primate brain: a long-term retrospective. Exp. Neurol. 210 (2008) 638-644
21. Kunwar S., Prados M.D., Chang S.M., Berger M.S., Lang F.F., Piepmeier J.M., Sampson J.H., Ram Z., Gutin P.H., Gibbons R.D., Aldape K.D., Croteau D.J., Sherman J.W., and Puri R.K. Direct intracerebral delivery of cintredekin besudotox (IL13-PE38QQR) in recurrent malignant glioma: a report by the Cintredekin Besudotox Intraparenchymal Study Group. J. Clin. Oncol. 25 (2007) 837-844
22. Lonser R.R., Walbridge S., Garmestani K., Butman J.A., Walters H.A., Vortmeyer A.O., Morrison P.F., Brechbiel M.W., and Oldfield E.H. Successful and safe perfusion of the primate brainstem: in vivo magnetic resonance imaging of macromolecular distribution during infusion. J. Neurosurg. 97 (2002) 905-913
23. Lonser R.R., Warren K.E., Butman J.A., Quezado Z., Robison R.A., Walbridge S., Schiffman R., Merrill M., Walker M.L., Park D.M., Croteau D., Brady R.O., and Oldfield E.H. Real-time image-guided direct convective perfusion of intrinsic brainstem lesions. Technical. note. J. Neurosurg. 107 (2007) 190-197
24. Mamot C., Drummond D.C., Hong K., Kirpotin D.B., and Park J.W. Liposome-based approaches to overcome anticancer drug resistance. Drug Resist. Updat. 6 (2003) 271-279
25. Mamot C., Nguyen J.B., Pourdehnad M., Hadaczek P., Saito R., Bringas J.R., Drummond D.C., Hong K., Kirpotin D.B., McKnight T., Berger M.S., Park J.W., and Bankiewicz K.S. Extensive distribution of liposomes in rodent brains and brain tumors following convection-enhanced delivery. J. Neurooncol. 68 (2004) 1-9
26. Matsushita T., Elliger S., Elliger C., Podsakoff G., Villarreal L., Kurtzman G.J., Iwaki Y., and Colosi P. Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther. 5 (1998) 938-945
27. Murad G.J., Walbridge S., Morrison P.F., Szerlip N., Butman J.A., Oldfield E.H., and Lonser R.R. Image-guided convection-enhanced delivery of gemcitabine to the brainstem. J. Neurosurg. 106 (2007) 351-356
28. Namavari M., Satyamurthy N., Phelps M., and Barrio J. Synthesis of 6-[18F] and 4-[18F]Fluoro-l-meta-tyrosines via regioselective radiofluorodestannylation. Appl. Radiat. Isotopes 44 (1993) 527
29. Neeves K.B., Sawyer A.J., Foley C.P., Saltzman W.M., and Olbricht W.L. Dilation and degradation of the brain extracellular matrix enhances penetration of infused polymer nanoparticles. Brain Res. 1180 (2007) 121-132
30. Nguyen J.B., Sanchez-Pernaute R., Cunningham J., and Bankiewicz K.S. Convection-enhanced delivery of AAV-2 combined with heparin increases TK gene transfer in the rat brain. Neuroreport 12 (2001) 1961-1964
31. Paxinos G., and Watson C. The Rat Brain in Stereotaxic Coordinates (1982), Academic Press, Sydney; New York
32. Saito R., Bringas J.R., McKnight T.R., Wendland M.F., Mamot C., Drummond D.C., Kirpotin D.B., Park J.W., Berger M.S., and Bankiewicz K.S. Distribution of liposomes into brain and rat brain tumor models by convection-enhanced delivery monitored with magnetic resonance imaging. Cancer Res. 64 (2004) 2572-2579
33. Saito R., Krauze M.T., Bringas J.R., Noble C., McKnight T.R., Jackson P., Wendland M.F., Mamot C., Drummond D.C., Kirpotin D.B., Hong K., Berger M.S., Park J.W., and Bankiewicz K.S. Gadolinium-loaded liposomes allow for real-time magnetic resonance imaging of convection-enhanced delivery in the primate brain. Exp. Neurol. 196 (2005) 381-389
34. Sampson J.H., Brady M.L., Petry N.A., Croteau D., Friedman A.H., Friedman H.S., Wong T., Bigner D.D., Pastan I., Puri R.K., and Pedain C. Intracerebral infusate distribution by convection-enhanced delivery in humans with malignant gliomas: descriptive effects of target anatomy and catheter positioning. Neurosurgery 60 (2007) ONS89-ONS98 discussion ONS98-89
35. Samulski R.J., Berns K.I., Tan M., and Muzyczka N. Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. Proc. Natl. Acad. Sci. U. S. A. 79 (1982) 2077-2081
36. Sanchez-Pernaute R., Harvey-White J., Cunningham J., and Bankiewicz K.S. Functional effect of adeno-associated virus mediated gene transfer of aromatic L-amino acid decarboxylase into the striatum of 6-OHDA-lesioned rats. Mol. Ther. 4 (2001) 324-330
37. Sanftner L.M., Sommer J.M., Suzuki B.M., Smith P.H., Vijay S., Vargas J.A., Forsayeth J.R., Cunningham J., Bankiewicz K.S., Kao H., Bernal J., Pierce G.F., and Johnson K.W. AAV2-mediated gene delivery to monkey putamen: evaluation of an infusion device and delivery parameters. Exp. Neurol. 194 (2005) 476-483
38. Stefanini M., De Martino C., and Zamboni L. Fixation of ejaculated spermatozoa for electron microscopy. Nature 216 (1967) 173-174
39. Sykova E. Extrasynaptic volume transmission and diffusion parameters of the extracellular space. Neuroscience 129 (2004) 861-876
40. Szerlip N.J., Walbridge S., Yang L., Morrison P.F., Degen J.W., Jarrell S.T., Kouri J., Kerr P.B., Kotin R., Oldfield E.H., and Lonser R.R. Real-time imaging of convection-enhanced delivery of viruses and virus-sized particles. J. Neurosurg. 107 (2007) 560-567
41. Urabe M., Ding C., and Kotin R.M. Insect cells as a factory to produce adeno-associated virus type 2 vectors. Hum. Gene Ther. 13 (2002) 1935-1943
42. Varenika V., Dickenson P., Bringas J., LeCouteur R., Higgins R., Park J.W., Fiandaca M.S., Berger M.S., Sampson J.H., and Bankiewicz K.S. Real-time imaging of CED in the brain permits detection of infusate leakage. J. Neurosurg. 109 (2008) 874-880
43. Vite C.H., Passini M.A., Haskins M.E., and Wolfe J.H. Adeno-associated virus vector-mediated transduction in the cat brain. Gene Ther. 10 (2003) 1874-1881
44. Wright J.F., Qu G., Tang C., and Sommer J.M. Recombinant adeno-associated virus: formulation challenges and strategies for a gene therapy vector. Curr. Opin. Drug Discov. Dev. 6 (2003) 174-178
45. Wu Z., Asokan A., and Samulski R.J. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol. Ther. 14 (2006) 316-327
46. Yin, D., Valles, F.E., Fiandaca, M.S., Forsayeth, J., Larson, P., Starr, P., Bankiewicz, K.S., in press. Striatal Volume Differences Between Non-human and Human Primates. J. Neurosci. Methods. doi:10.1016/jneumeth.2008.08.027.