Delivery of therapeutic agents through intracerebroventricular (ICV) and intravenous (IV) injection in mice

Journal of Visualized Experiments

Volumn , Issue 56, 2011, Pages

  Glascock, Jacqueline J ^a   Osman, Erkan Y ^a   Coady, Tristan H ^b   Rose, Ferrill F ^a   Shababi, Monir ^a   Lorson, Christian L ^a  

^a UNIVERSITY OF MISSOURI   (United States)

^b Och Spine at New York Presbyterian Hospitals   (United States)

Author keywords

Brain; CNS; Injection; Issue 56; Medicine; Motor neuron; Mouse; Neuroscience; Temporal vein; Ventricles

Indexed keywords

EID: 80054879102     PISSN: 1940087X     EISSN: None     Source Type: Journal    
DOI: 10.3791/2968     Document Type: Article

References (17)

1. Blanchette, M. & Fortin, D. Blood-brain barrier disruption in the treatment of brain tumors. Methods Mol. Biol. 686, 447-463 (2011).
2. Foust, K.D. & Kaspar, B.K. Over the barrier and through the blood: to CNS delivery we go. Cell Cycle. 24 (8), 4017-4018 (2009).
3. Snyder, E.Y., Taylor, R.M., & Wolfe, J.H. Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain. Nature. 374 (6520), 367-370 (1995).
4. Passini, M.A. & Wolfe, J.H. Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector. J. Virol. 24 (75), 12382-12392 (2001).
5. Kienstra, K.A., Freysdottir, D., Gonzales, N.M., & Hirschi, K.K. Murine neonatal intravascular injections: modeling newborn disease. J. Am. Assoc. Lab. Anim. Sci. 46 (6), 50-54 (2007).
6. Sands, M.S. & Barker, J.E. Percutaneous intravenous injection in neonatal mice. Lab. Anim. Sci. 49 (3), 328-330 (1999).
7. Foust, K.D., et al. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat. Biotechnol. 28 (3), 271-274 (2010).
8. Foust, K.D. et al. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat. Biotechnol. 27 (1), 59-65 (2009).
9. Passini, M.A., Watson, D.J., & Wolfe, J.H. Gene delivery to the mouse brain with adeno-associated virus. Methods Mol. Biol. 246, 225-236 (2004).
10. Coady, T.H. & Lorson, C.L. Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy. J. Neurosci. 30 (1), 126-130 (2010).
11. Baughan, T.D., Dickson, A., Osman, E.Y., & Lorson, C.L. Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy. Hum. Mol. Genet. 18 (9), 1600-1611 (2009).
12. Coady, T.H., Baughan, T.D., Shababi, M., Passini, M.A., & Lorson, C.L. Development of a single vector system that enhances trans-splicing of SMN2 transcripts. PLoS One. 3 (10), e3468 (2008).
13. Dickson, A., Osman, E., & Lorson, C.A Negatively-Acting Bifunctional RNA Increases Survival Motor Neuron in vitro and in vivo. Hum. Gene. Ther. 19 (11), 1307-1315(2008).
14. Mattis, V.B., Ebert, A.D., Fosso, M.Y., Chang, C.W., & Lorson, C.L. Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model. Hum. Mol. Genet. 18 (20), 3906-3913 (2009).
15. Williams, J.H., et al. Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy. J. Neurosci. 29 (24), 7633-7638 (2009).
16. Passini, M.A., et al. CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy. J. Clin. Invest. 120(4), 1253-1264 (2010).
17. Shababi, M., Glascock, J., & Lorson, C.L., Combination of SMN Trans-Splicing and a Neurotrophic Factor Increases the Life Span and Body Mass in a Severe Model of Spinal Muscular Atrophy. Hum. Gene. Ther. 22, 1-10 (2010).