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Items: 1 to 20 of 157


Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders.

Bevan AK, Duque S, Foust KD, Morales PR, Braun L, Schmelzer L, Chan CM, McCrate M, Chicoine LG, Coley BD, Porensky PN, Kolb SJ, Mendell JR, Burghes AH, Kaspar BK.

Mol Ther. 2011 Nov;19(11):1971-80. doi: 10.1038/mt.2011.157.


Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.

Meyer K, Ferraiuolo L, Schmelzer L, Braun L, McGovern V, Likhite S, Michels O, Govoni A, Fitzgerald J, Morales P, Foust KD, Mendell JR, Burghes AH, Kaspar BK.

Mol Ther. 2015 Mar;23(3):477-87. doi: 10.1038/mt.2014.210.


Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice.

Benkhelifa-Ziyyat S, Besse A, Roda M, Duque S, Astord S, Carcenac R, Marais T, Barkats M.

Mol Ther. 2013 Feb;21(2):282-90. doi: 10.1038/mt.2012.261.


The advent of AAV9 expands applications for brain and spinal cord gene delivery.

Dayton RD, Wang DB, Klein RL.

Expert Opin Biol Ther. 2012 Jun;12(6):757-66. doi: 10.1517/14712598.2012.681463. Review.


Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates.

Gray SJ, Nagabhushan Kalburgi S, McCown TJ, Jude Samulski R.

Gene Ther. 2013 Apr;20(4):450-9. doi: 10.1038/gt.2012.101. Erratum in: Gene Ther. 2013 Apr;20(4):465.


Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy.

Passini MA, Bu J, Richards AM, Treleaven CM, Sullivan JA, O'Riordan CR, Scaria A, Kells AP, Samaranch L, San Sebastian W, Federici T, Fiandaca MS, Boulis NM, Bankiewicz KS, Shihabuddin LS, Cheng SH.

Hum Gene Ther. 2014 Jul;25(7):619-30. doi: 10.1089/hum.2014.011.


Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats.

Bucher T, Dubreil L, Colle MA, Maquigneau M, Deniaud J, Ledevin M, Moullier P, Joussemet B.

Gene Ther. 2014 May;21(5):522-8. doi: 10.1038/gt.2014.16.


Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes.

Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK.

Nat Biotechnol. 2009 Jan;27(1):59-65. doi: 10.1038/nbt.1515.


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

Dirren E, Towne CL, Setola V, Redmond DE Jr, Schneider BL, Aebischer P.

Hum Gene Ther. 2014 Feb;25(2):109-20. doi: 10.1089/hum.2013.021.


Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates.

Samaranch L, Salegio EA, San Sebastian W, Kells AP, Bringas JR, Forsayeth J, Bankiewicz KS.

Hum Gene Ther. 2013 May;24(5):526-32. doi: 10.1089/hum.2013.005.


Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs.

Federici T, Taub JS, Baum GR, Gray SJ, Grieger JC, Matthews KA, Handy CR, Passini MA, Samulski RJ, Boulis NM.

Gene Ther. 2012 Aug;19(8):852-9. doi: 10.1038/gt.2011.130.


Intrathecal shRNA-AAV9 inhibits target protein expression in the spinal cord and dorsal root ganglia of adult mice.

Hirai T, Enomoto M, Machida A, Yamamoto M, Kuwahara H, Tajiri M, Hirai Y, Sotome S, Mizusawa H, Shinomiya K, Okawa A, Yokota T.

Hum Gene Ther Methods. 2012 Apr;23(2):119-27. doi: 10.1089/hgtb.2012.035.


Comparison of adeno-associated viral vector serotypes for spinal cord and motor neuron gene delivery.

Snyder BR, Gray SJ, Quach ET, Huang JW, Leung CH, Samulski RJ, Boulis NM, Federici T.

Hum Gene Ther. 2011 Sep;22(9):1129-35. doi: 10.1089/hum.2011.008.


PTEN depletion decreases disease severity and modestly prolongs survival in a mouse model of spinal muscular atrophy.

Little D, Valori CF, Mutsaers CA, Bennett EJ, Wyles M, Sharrack B, Shaw PJ, Gillingwater TH, Azzouz M, Ning K.

Mol Ther. 2015 Feb;23(2):270-7. doi: 10.1038/mt.2014.209.


Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors.

Miyake N, Miyake K, Yamamoto M, Hirai Y, Shimada T.

Brain Res. 2011 May 10;1389:19-26. doi: 10.1016/j.brainres.2011.03.014.


Widespread neuron-specific transgene expression in brain and spinal cord following synapsin promoter-driven AAV9 neonatal intracerebroventricular injection.

McLean JR, Smith GA, Rocha EM, Hayes MA, Beagan JA, Hallett PJ, Isacson O.

Neurosci Lett. 2014 Jul 25;576:73-8. doi: 10.1016/j.neulet.2014.05.044.


scAAV9 intracisternal delivery results in efficient gene transfer to the central nervous system of a feline model of motor neuron disease.

Bucher T, Colle MA, Wakeling E, Dubreil L, Fyfe J, Briot-Nivard D, Maquigneau M, Raoul S, Cherel Y, Astord S, Duque S, Marais T, Voit T, Moullier P, Barkats M, Joussemet B.

Hum Gene Ther. 2013 Jul;24(7):670-82. doi: 10.1089/hum.2012.218.


Intravascular AAV9 Administration for Delivering RNA Silencing Constructs to the CNS and Periphery.

Dufour BD, McBride JL.

Methods Mol Biol. 2016;1364:261-75. doi: 10.1007/978-1-4939-3112-5_21.


Systemic delivery of tyrosine-mutant AAV vectors results in robust transduction of neurons in adult mice.

Iida A, Takino N, Miyauchi H, Shimazaki K, Muramatsu S.

Biomed Res Int. 2013;2013:974819. doi: 10.1155/2013/974819.

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