Intracerebroventricular injection of adeno-associated virus 6 and 9 vectors for cell type-specific transgene expression in the spinal cord

Human Gene Therapy

Volumn 25, Issue 2, 2014, Pages 109-120

  Dirren, Elisabeth ^a   Towne, Christopher L ^a   Setola, Veronica ^a   Redmond, Donald E ^b   Schneider, Bernard L ^a   Aebischer, Patrick ^a  

^a EPFL   (Switzerland)

^b YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS 6 VECTOR; ADENOVIRUS 9 VECTOR; ADENOVIRUS VECTOR; UNCLASSIFIED DRUG;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; ASTROCYTE; CEREBROSPINAL FLUID; CISTERNA MAGNA; CONTROLLED STUDY; CYTOMEGALOVIRUS; GENE EXPRESSION; MOTONEURON; MOTOR NEURON DISEASE; MOUSE; NONHUMAN; PRIMATE; SPINAL CORD; SPINAL CORD MOTONEURON; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; ASTROCYTES; CERCOPITHECUS AETHIOPS; DEPENDOVIRUS; GENE DOSAGE; GENE EXPRESSION; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; MALE; MICE; MOTOR NEURONS; ORGAN SPECIFICITY; PROMOTER REGIONS, GENETIC; SPINAL CORD; TRANSDUCTION, GENETIC; TRANSGENES;

EID: 84893586080     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2013.021     Document Type: Article

References (32)

1. Andersen, P.M., and Al-Chalabi, A. (2011). Clinical genetics of amyotrophic lateral sclerosis: what do we really know? Nat. Rev. Neurol. 7, 603-615.
2. Bevan, A.K., Duque, S., Foust, K.D., et al. (2011). Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders. Mol. Ther. 19, 1971-1980.
3. Boillee, S., Yamanaka, K., Lobsiger, C.S., et al. (2006). Onset and progression in inherited ALS determined by motor neurons and microglia. Science 312, 1389-1392.
4. Clement, A.M., Nguyen, M.D., Roberts, E.A., et al. (2003). Wildtype nonneuronal cells extend survival of SOD1 mutant motor neurons in ALS mice. Science 302, 113-117.
5. Duque, S., Joussemet, B., Riviere, C., et al. (2009). Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol. Ther. 17, 1187-1196.
6. Dusonchet, J., Bensadoun, J.C., Schneider, B.L., and Aebischer, P. (2009). Targeted overexpression of the parkin substrate Pael-R in the nigrostriatal system of adult rats to model Parkinson's disease. Neurobiol. Dis. 35, 32-41.
7. Federici, T., and Boulis, N.M. (2012). Gene therapy for amyotrophic lateral sclerosis. Neurobiol. Dis. 48, 236-242.
8. Federici, T., Taub, J.S., Baum, G.R., et al. (2011). Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs. Gene Ther. 19, 852-859.
9. Foust, K.D., Nurre, E., Montgomery, C.L., et al. (2009). Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat. Biotechnol. 27, 59-65.
10. Gray, S.J., Matagne, V., Bachaboina, L., et al. (2011). Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol. Ther. 19, 1058-1069.
11. Inagaki, K., Fuess, S., Storm, T.A., et al. (2006). Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. Mol. Ther. 14, 45-53.
12. Ince, P.G., Highley, J.R., Kirby, J., et al. (2011). Molecular pathology and genetic advances in amyotrophic lateral sclerosis: an emerging molecular pathway and the significance of glial pathology. Acta Neuropathol. 122, 657-671.
13. Koerber, J.T., Klimczak, R., Jang, J.H., et al. (2009). Molecular evolution of adeno-associated virus for enhanced glial gene delivery. Mol. Ther. 17, 2088-2095.
14. Kotchey, N.M., Adachi, K., Zahid, M., et al. (2011). A potential role of distinctively delayed blood clearance of recombinant adeno-associated virus serotype 9 in robust cardiac transduction. Mol. Ther. 19, 1079-1089.
15. Lawlor, P.A., Bland, R.J., Mouravlev, A., et al. (2009). Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates. Mol. Ther. 17, 1692-1702.
16. Lee, Y., Messing, A., Su, M., and Brenner, M. (2008). GFAP promoter elements required for region-specific and astrocytespecific expression. Glia 56, 481-493.
17. McCarty, D.M., Fu, H., Monahan, P.E., et al. (2003). Adenoassociated virus terminal repeat (TR) mutant generates selfcomplementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther. 10, 2112-2118.
18. Mingozzi, F., and High, K.A. (2011). Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat. Rev. Genet. 12, 341-355.
19. Miyake, N., Miyake, K., Yamamoto, M., et al. (2011). Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors. Brain Res. 1389, 19-26.
20. Passini, M.A., and Cheng, S.H. (2011). Prospects for the gene therapy of spinal muscular atrophy. Trends Mol. Med. 17, 259-265.
21. Passini, M.A., Bu, J., Roskelley, E.M., et al. (2010). CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy. J. Clin. Invest. 120, 1253-1264.
22. Peviani, M., Kurosaki, M., Terao, M., et al. (2012). Lentiviral vectors carrying enhancer elements of Hb9 promoter drive selective transgene expression in mouse spinal cord motor neurons. J. Neurosci. Methods 205, 139-147.
23. Robberecht, W., and Philips, T. (2013). The changing scene of amyotrophic lateral sclerosis. Nat. Rev. Neurosci. 14, 248-264.
24. Samaranch, L., Salegio, E.A., San Sebastian, W., et al. (2012). Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Hum. Gene Ther. 23, 382-389.
25. Snyder, B.R., Gray, S.J., Quach, E.T., et al. (2011). Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery. Hum. Gene Ther. 22, 1129-1135.
26. Towne, C., Schneider, B.L., Kieran, D., et al. (2010). Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6. Gene Ther. 17, 141-146.
27. Towne, C., Setola, V., Schneider, B.L., and Aebischer, P. (2011). Neuroprotection by gene therapy targeting mutant SOD1 in individual pools of motor neurons does not translate into therapeutic benefit in fALS mice. Mol. Ther. 19, 274-283.
28. Van Blitterswijk, M., Dejesus-Hernandez, M., and Rademakers, R. (2012). How do C9ORF72 repeat expansions cause amyotrophic lateral sclerosis and frontotemporal dementia: can we learn from other noncoding repeat expansion disorders? Curr. Opin. Neurol. 25, 689-700.
29. Wang, Z., Ma, H.I., Li, J., et al. (2003). Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther. 10, 2105-2111.
30. Wu, Z., Asokan, A., and Samulski, R.J. (2006). Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol. Ther. 14, 316-327.
31. Yamanaka, K., Chun, S.J., Boillee, S., et al. (2008). Astrocytes as determinants of disease progression in inherited amyotrophic lateral sclerosis. Nat. Neurosci. 11, 251-253.
32. Zhang, H., Yang, B., Mu, X., et al. (2011). Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol. Ther. 19, 1440-1448.