Intra-arterial delivery of AAV vectors to the mouse brain after mannitol mediated blood brain barrier disruption

Journal of Controlled Release

Volumn 196, Issue , 2014, Pages 71-78

  Foley, Conor P ^a   Rubin, David G ^b   Santillan, Alejandro ^b   Sondhi, Dolan ^b   Dyke, Jonathan P ^a   Pierre Gobin, Y ^b   Crystal, Ronald G ^b   Ballon, Douglas J ^a,b  

^a CITIGROUP   (United States)

^b WEILL CORNELL MEDICAL COLLEGE   (United States)

Author keywords

Adeno associated virus; Blood brain barrier; Intra arterial; MRI; Neural drug delivery

Indexed keywords

AAV VECTORS; ADENO-ASSOCIATED VIRUS; BLOOD-BRAIN BARRIER; INTRA-ARTERIAL; MOUSE BRAIN; NEURAL DRUG DELIVERIES;

MAGNETIC RESONANCE IMAGING;

CONTRAST MEDIUM; MANNITOL; PARVOVIRUS VECTOR; DIURETIC AGENT;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ANTERIOR CEREBRAL ARTERY; ANTERIOR CHOROIDAL CEREBRAL ARTERY; ARTICLE; BLOOD BRAIN BARRIER; BRAIN; BRAIN ARTERY; CLN2 GENE; CONTRAST ENHANCEMENT; CONTROLLED STUDY; DRUG DELIVERY SYSTEM; ENZYME ACTIVITY; GENE; GENE THERAPY; HEART; IMMUNOFLUORESCENCE; IMMUNOHISTOCHEMISTRY; KIDNEY; LIVER; LUNG; LYSOSOME STORAGE DISEASE; MALE; MIDDLE CEREBRAL ARTERY; MOUSE; NERVOUS TISSUE; NEURONAL CEROID LIPOFUSCINOSIS; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; QUADRICEPS FEMORIS MUSCLE; SPLEEN; TRANSGENE; VIRUS PARTICLE; ANATOMY AND HISTOLOGY; ANIMAL; CATHETER; DEPENDOPARVOVIRUS; DRUG EFFECTS; GENE TRANSFER; GENE VECTOR; INTRAARTERIAL DRUG ADMINISTRATION; OSMOSIS; TISSUE DISTRIBUTION; VIROLOGY;

ANIMALS; BLOOD-BRAIN BARRIER; BRAIN; CATHETERS; DEPENDOVIRUS; DIURETICS; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; INJECTIONS, INTRA-ARTERIAL; MAGNETIC RESONANCE IMAGING; MALE; MANNITOL; MICE; OSMOSIS; TISSUE DISTRIBUTION;

EID: 84908191350     PISSN: 01683659     EISSN: 18734995     Source Type: Journal    
DOI: 10.1016/j.jconrel.2014.09.018     Document Type: Article

References (31)

1. W.M. Pardridge Drug and gene delivery to the brain: the vascular route Neuron 36 Nov 2002 555 558
2. D.H. Abramson, I.J. Dunkel, S.E. Brodie, J.W. Kim, and Y.P. Gobin A phase I/II study of direct intraarterial (ophthalmic artery) chemotherapy with melphalan for intraocular retinoblastoma initial results Ophthalmology 115 Aug 2008 1398 1404 (1404.e1)
3. K.L. Black, T. Cloughesy, S.C. Huang, Y.P. Gobin, Y. Zhou, J. Grous, G. Nelson, K. Farahani, C.K. Hoh, and M. Phelps Intracarotid infusion of RMP-7, a bradykinin analog, and transport of gallium-68 ethylenediamine tetraacetic acid into human gliomas J. Neurosurg. 86 Apr 1997 603 609
4. T.F. Cloughesy, Y.P. Gobin, K.L. Black, F. Viñuela, F. Taft, B. Kadkhoda, and F. Kabbinavar Intra-arterial carboplatin chemotherapy for brain tumors: a dose escalation study based on cerebral blood flow J. Neurooncol. 35 Nov 1997 121 131
5. C. Aliberti, G. Benea, M. Tilli, and G. Fiorentini Chemoembolization (TACE) of unresectable intrahepatic cholangiocarcinoma with slow-release doxorubicin-eluting beads: preliminary results Cardiovasc. Intervent. Radiol. 31 5 2008 883 888
6. J. Kettenbach, A. Stadler, I.V. Katzler, R. Schernthaner, M. Blum, J. Lammer, and T. Rand Drug-loaded microspheres for the treatment of liver cancer: review of current results Cardiovasc. Intervent. Radiol. 31 3 2008 468 476
7. R. Jahan, G.R. Duckwiler, C.S. Kidwell, J.W. Sayre, Y.P. Gobin, J.P. Villablanca, J. Saver, S. Starkman, N. Martin, and F. Vinuela Intra-arterial thrombolysis for treatment of acute stroke: experience in 26 patients with long-term follow-up AJNR Am. J. Neuroradiol. 20 Aug 1999 1291 1299
8. N.D. Doolittle, M.E. Miner, W.A. Hall, T. Siegal, E. Jerome, E. Osztie, L.D. McAllister, J.S. Bubalo, D.F. Kraemer, D. Fortin, R. Nixon, L.L. Muldoon, and E.A. Neuwelt Safety and efficacy of a multicenter study using intraarterial chemotherapy in conjunction with osmotic opening of the blood-brain barrier for the treatment of patients with malignant brain tumors Cancer 88 Feb 2000 637 647
9. D. Fortin, C. Gendron, M. Boudrias, and M.-P. Garant Enhanced chemotherapy delivery by intraarterial infusion and blood-brain barrier disruption in the treatment of cerebral metastasis Cancer 109 Feb 2007 751 760
10. N.G. Rainov, K. Ikeda, N.H. Qureshi, S. Grover, U. Herrlinger, P. Pechan, E.A. Chiocca, X.O. Breakefield, and F.H. Barnett Intraarterial delivery of adenovirus vectors and liposome-DNA complexes to experimental brain neoplasms Hum. Gene Ther. 10 Jan 1999 311 318
11. W.E. Zink, C.P. Foley, J.P. Dyke, M.J. Synan, A.L. Chakrapani, D.J. Ballon, W.L. Olbricht, and Y.P. Gobin Novel microcatheters for selective intra-arterial injection of fluid in the rat brain AJNR Am. J. Neuroradiol. 30 Jun 2009 1190 1196
12. D. Sondhi, N.R. Hackett, D.A. Peterson, J. Stratton, M. Baad, K.M. Travis, J.M. Wilson, and R.G. Crystal Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector Mol. Ther. 15 Mar 2007 481 491
13. D. Sondhi, D.A. Peterson, E.L. Giannaris, C.T. Sanders, B.S. Mendez, B. De, A.B. Rostkowski, B. Blanchard, K. Bjugstad, J.R. Sladek, D.E. Redmond, P.L. Leopold, S.M. Kaminsky, N.R. Hackett, and R.G. Crystal AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL Gene Ther. 12 Nov 2005 1618 1632
14. T.M. Daly, C. Vogler, B. Levy, M.E. Haskins, and M.S. Sands Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease Proc. Natl. Acad. Sci. U. S. A. 96 Mar 1999 2296 2300
15. L. Lin, and P. Lobel Production and characterization of recombinant human CLN2 protein for enzyme-replacement therapy in late infantile neuronal ceroid lipofuscinosis Biochem. J. 357 Jul 2001 49 55
16. A. Santillan, D.G. Rubin, C.P. Foley, D. Sondhi, R.G. Crystal, Y.P. Gobin, and D. Ballon Cannulation of the internal carotid artery in mice: a novel technique for intra-arterial delivery of therapeutics J. Neurosci. Methods 222 2014 106 110
17. W.C. Cosolo, P. Martinello, W.J. Louis, and N. Christophidis Blood-brain barrier disruption using mannitol: time course and electron microscopy studies Am. J. Physiol. 256 Feb 1989 R443 R447
18. M.D. Abramoff, P.J. Magelhaes, and S.J. Ram Image processing with ImageJ Biophoton. Int. 11 7 2004 36 42
19. D.M. McCarty, J. DiRosario, K. Gulaid, J. Muenzer, and H. Fu Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice Gene Ther. 16 Nov 2009 1340 1352
20. M.A. Passini, J.C. Dodge, J. Bu, W. Yang, Q. Zhao, D. Sondhi, N.R. Hackett, S.M. Kaminsky, Q. Mao, L.S. Shihabuddin, S.H. Cheng, D.E. Sleat, G.R. Stewart, B.L. Davidson, P. Lobel, and R.G. Crystal Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis J. Neurosci. 26 Feb 2006 1334 1342
21. I. Sohar, L. Lin, and P. Lobel Enzyme-based diagnosis of classical late infantile neuronal ceroid lipofuscinosis: comparison of tripeptidyl peptidase I and pepstatin-insensitive protease assays Clin. Chem. 46 Jul 2000 1005 1008
22. M. Takeda, Y. Katayama, T. Tsutsui, T. Komeyama, and T. Mizusawa Does gadolinium-diethylene triamine pentaacetic acid enhanced MRI of kidney represent tissue concentration of contrast media in the kidney? In vivo and in vitro study Magn. Reson. Imaging 12 3 1994 421 427
23. E.A. Neuwelt, D.L. Goldman, S.A. Dahlborg, J. Crossen, F. Ramsey, S. Roman-Goldstein, R. Braziel, and B. Dana Primary CNS lymphoma treated with osmotic blood-brain barrier disruption: prolonged survival and preservation of cognitive function J. Clin. Oncol. 9 Sep 1991 1580 1590
24. S. Joshi, A. Ergin, M. Wang, R. Reif, J. Zhang, J.N. Bruce, and I.J. Bigio Inconsistent blood brain barrier disruption by intraarterial mannitol in rabbits: implications for chemotherapy J. Neurooncol. 104 Aug 2011 11 19
25. E.A. Neuwelt, K.R. Maravilla, E.P. Frenkel, S.I. Rapaport, S.A. Hill, and P.A. Barnett Osmotic blood-brain barrier disruption. Computerized tomographic monitoring of chemotherapeutic agent delivery J. Clin. Invest. 64 Aug 1979 684 688
26. E.A. Neuwelt, E.P. Frenkel, J. Diehl, L.H. Vu, S. Rapoport, and S. Hill Reversible osmotic blood-brain barrier disruption in humans: implications for the chemotherapy of malignant brain tumors Neurosurgery 7 Jul 1980 44 52
27. M. Blanchette, M. Pellerin, L. Tremblay, M. Lepage, and D. Fortin Real-time monitoring of gadolinium diethylenetriamine penta-acetic acid during osmotic blood-brain barrier disruption using magnetic resonance imaging in normal Wistar rats Neurosurgery 65 Aug 2009 344 350 (discussion 350-1)
28. L. Chen, K.R. Swartz, and M. Toborek Vessel microport technique for applications in cerebrovascular research J. Neurosci. Res. 87 May 2009 1718 1727
29. CUhCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates Hum. Gene Ther. 16 Dec 2005 1484 1503
30. D. Sondhi, L. Johnson, K. Purpura, S. Monette, M. Souweidane, M.G. Kaplitt, B. Kosofsky, K. Yohay, D. Ballon, J. Dyke, S.M. Kaminksy, N.R. Hackett, and R.G. Crystal Long term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and non-human primates for the treatment of late infantile neuronal ceroid lipofuscinosis Hum. Gene Ther. Methods 23 Oct 2012 324 335
31. D.E. Oken Renal and extrarenal considerations in high-dose mannitol therapy Ren. Fail. 16 1 1994 147 159