Abstract
Gene therapy has emerged as a potential avenue of treatment for many neurological disorders. Technological advances in imaging techniques allow for the monitoring of real-time infusions into the brain of rodents, nonhuman primates, and humans. Here, we discuss the use of magnetic resonance imaging (MRI) as a tool in the delivery of adeno-associated viral (AAV) particles into brain of nonhuman primates.
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References
Chen Y, Liu L (2012) Modern methods for delivery of drugs across the blood-brain barrier. Adv Drug Deliv Rev 64(7):640–665
Salvatore MF, Ai Y, Fischer B et al (2006) Point source concentration of GDNF may explain failure of phase II clinical trial. Exp Neurol 202(2):497–505
Marks WJ Jr, Ostrem JL, Verhagen L 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(5):400–408
Eberling JL, Kells AP, Pivirotto P et al (2009) Functional effects of AAV2-GDNF on the dopaminergic nigrostriatal pathway in parkinsonian rhesus monkeys. Hum Gene Ther 20(5):511–518
Bartus RT, Herzog CD, Chu Y et al (2011) Bioactivity of AAV2-neurturin gene therapy (CERE-120): differences between Parkinson’s disease and nonhuman primate brains. Mov Disord 26(1):27–36
Marks WJ Jr, Bartus RT, Siffert J et al (2010) Gene delivery of AAV2-neurturin for Parkinson’s disease: a double-blind, randomised, controlled trial. Lancet Neurol 9(12):1164–1172
Richardson R, Gimenez F, Salegio EA et al (2011) T2-imaging in monitoring of intraparenchymal real-time convection enhanced delivery. Neurosurgery 69(1):154–163
Richardson RM, Kells AP, Martin AJ et al (2011) Novel platform for MRI-guided convection-enhanced delivery of therapeutics: preclinical validation in nonhuman primate brain. Stereotact Funct Neurosurg 89(3):141–151
San Sebastian W, Richardson RM, Kells AP et al (2012) Safety and tolerability of magnetic resonance imaging-guided convection-enhanced delivery of AAV2-hAADC with a novel delivery platform in nonhuman primate striatum. Hum Gene Ther 23(2):210–217
Salegio E, Kells AP, Richardson RM et al (2010) Magnetic resonance imaging-guided delivery of adeno-associated virus type 2 to the primate brain for the treatment of lysosomal storage disorders. Hum Gene Ther 21:1–11
Hadaczek P, Kohutnicka M, Krauze MT et al (2006) Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain. Hum Gene Ther 17(3):291–302
Yin D, Richardson R, Fiandaca M et al (2010) Cannula placement for effective convection-enhanced delivery in the nonhuman primate thalamus and brainstem: implications for clinical delivery of therapeutics. J Neurosurg 113:240–248
Paxinos G, Huang X, Petrides M, Toga A (2008) The rhesus monkey brain. Elsevier Inc, USA
Yin D, Forsayeth J, Bankiewicz K (2010) Optimized cannula design and placement for convection-enhanced delivery in rat striatum. J Neurosci Methods 178:46–51
Krauze MT, Saito R, Noble C et al (2005) Reflux-free cannula for convection-enhanced high-speed delivery of therapeutic agents. J Neurosurg 103(5):923–929
Acknowledgement
This work was supported by a grant to K.S.B. from NIH-NINDS (R01NS073940-01).
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Salegio, E.A., Bringas, J., Bankiewicz, K.S. (2016). MRI-Guided Delivery of Viral Vectors. In: Manfredsson, F. (eds) Gene Therapy for Neurological Disorders. Methods in Molecular Biology, vol 1382. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-3271-9_15
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DOI: https://doi.org/10.1007/978-1-4939-3271-9_15
Publisher Name: Humana Press, New York, NY
Print ISBN: 978-1-4939-3270-2
Online ISBN: 978-1-4939-3271-9
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