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Items: 34


Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK.

N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198.


Systemic gene delivery transduces the enteric nervous system of guinea pigs and cynomolgus macaques.

Gombash SE, Cowley CJ, Fitzgerald JA, Lepak CA, Neides MG, Hook K, Todd LJ, Wang GD, Mueller C, Kaspar BK, Bielefeld EC, Fischer AJ, Wood JD, Foust KD.

Gene Ther. 2017 Oct;24(10):640-648. doi: 10.1038/gt.2017.72. Epub 2017 Aug 3.


Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3).

Bosch ME, Aldrich A, Fallet R, Odvody J, Burkovetskaya M, Schuberth K, Fitzgerald JA, Foust KD, Kielian T.

J Neurosci. 2016 Sep 14;36(37):9669-82. doi: 10.1523/JNEUROSCI.1635-16.2016.


Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis.

Song S, Miranda CJ, Braun L, Meyer K, Frakes AE, Ferraiuolo L, Likhite S, Bevan AK, Foust KD, McConnell MJ, Walker CM, Kaspar BK.

Nat Med. 2016 Apr;22(4):397-403. doi: 10.1038/nm.4052. Epub 2016 Feb 29.


Systemic Gene Therapy for Targeting the CNS.

Gombash SE, Foust KD.

Methods Mol Biol. 2016;1382:231-7. doi: 10.1007/978-1-4939-3271-9_16.


Low levels of Survival Motor Neuron protein are sufficient for normal muscle function in the SMNΔ7 mouse model of SMA.

Iyer CC, McGovern VL, Murray JD, Gombash SE, Zaworski PG, Foust KD, Janssen PM, Burghes AH.

Hum Mol Genet. 2015 Nov 1;24(21):6160-73. doi: 10.1093/hmg/ddv332. Epub 2015 Aug 13.


SMN deficiency disrupts gastrointestinal and enteric nervous system function in mice.

Gombash SE, Cowley CJ, Fitzgerald JA, Iyer CC, Fried D, McGovern VL, Williams KC, Burghes AH, Christofi FL, Gulbransen BD, Foust KD.

Hum Mol Genet. 2015 Oct 1;24(19):5665. doi: 10.1093/hmg/ddv292. Epub 2015 Jul 29. No abstract available.


SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA.

McGovern VL, Iyer CC, Arnold WD, Gombash SE, Zaworski PG, Blatnik AJ 3rd, Foust KD, Burghes AH.

Hum Mol Genet. 2015 Oct 1;24(19):5524-41. doi: 10.1093/hmg/ddv283. Epub 2015 Jul 23.


Endolysosomal Deficits Augment Mitochondria Pathology in Spinal Motor Neurons of Asymptomatic fALS Mice.

Xie Y, Zhou B, Lin MY, Wang S, Foust KD, Sheng ZH.

Neuron. 2015 Jul 15;87(2):355-70. doi: 10.1016/j.neuron.2015.06.026.


SMN deficiency disrupts gastrointestinal and enteric nervous system function in mice.

Gombash SE, Cowley CJ, Fitzgerald JA, Iyer CC, Fried D, McGovern VL, Williams KC, Burghes AH, Christofi FL, Gulbransen BD, Foust KD.

Hum Mol Genet. 2015 Jul 1;24(13):3847-60. doi: 10.1093/hmg/ddv127. Epub 2015 Apr 9. Erratum in: Hum Mol Genet. 2015 Oct 1;24(19):5665.


Gene delivery to the spinal cord using MRI-guided focused ultrasound.

Weber-Adrian D, Thévenot E, O'Reilly MA, Oakden W, Akens MK, Ellens N, Markham-Coultes K, Burgess A, Finkelstein J, Yee AJ, Whyne CM, Foust KD, Kaspar BK, Stanisz GJ, Chopra R, Hynynen K, Aubert I.

Gene Ther. 2015 Jul;22(7):568-77. doi: 10.1038/gt.2015.25. Epub 2015 Apr 23.


Intravenous injections in neonatal mice.

Gombash Lampe SE, Kaspar BK, Foust KD.

J Vis Exp. 2014 Nov 11;(93):e52037. doi: 10.3791/52037.


Intravenous AAV9 efficiently transduces myenteric neurons in neonate and juvenile mice.

Gombash SE, Cowley CJ, Fitzgerald JA, Hall JC, Mueller C, Christofi FL, Foust KD.

Front Mol Neurosci. 2014 Oct 15;7:81. doi: 10.3389/fnmol.2014.00081. eCollection 2014.


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. Epub 2014 Oct 31.


Microglia induce motor neuron death via the classical NF-κB pathway in amyotrophic lateral sclerosis.

Frakes AE, Ferraiuolo L, Haidet-Phillips AM, Schmelzer L, Braun L, Miranda CJ, Ladner KJ, Bevan AK, Foust KD, Godbout JP, Popovich PG, Guttridge DC, Kaspar BK.

Neuron. 2014 Mar 5;81(5):1009-1023. doi: 10.1016/j.neuron.2014.01.013.


Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS.

Foust KD, Salazar DL, Likhite S, Ferraiuolo L, Ditsworth D, Ilieva H, Meyer K, Schmelzer L, Braun L, Cleveland DW, Kaspar BK.

Mol Ther. 2013 Dec;21(12):2148-59. doi: 10.1038/mt.2013.211. Epub 2013 Sep 6.


Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome.

Garg SK, Lioy DT, Cheval H, McGann JC, Bissonnette JM, Murtha MJ, Foust KD, Kaspar BK, Bird A, Mandel G.

J Neurosci. 2013 Aug 21;33(34):13612-20. doi: 10.1523/JNEUROSCI.1854-13.2013.


Targeted delivery of self-complementary adeno-associated virus serotype 9 to the brain, using magnetic resonance imaging-guided focused ultrasound.

Thévenot E, Jordão JF, O'Reilly MA, Markham K, Weng YQ, Foust KD, Kaspar BK, Hynynen K, Aubert I.

Hum Gene Ther. 2012 Nov;23(11):1144-55. doi: 10.1089/hum.2012.013. Epub 2012 Oct 15.


Glutamate Transporter GLT-1 Upregulation Attenuates Visceral Nociception and Hyperalgesia via Spinal Mechanisms Not Related to Anti-Inflammatory or Probiotic Effects.

Lin Y, Roman K, Foust KD, Kaspar BK, Bailey MT, Stephens RL.

Pain Res Treat. 2011;2011:507029. doi: 10.1155/2011/507029. Epub 2011 Dec 12.


Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates.

Samaranch L, Salegio EA, San Sebastian W, Kells AP, Foust KD, Bringas JR, Lamarre C, Forsayeth J, Kaspar BK, Bankiewicz KS.

Hum Gene Ther. 2012 Apr;23(4):382-9. doi: 10.1089/hum.2011.200. Epub 2012 Mar 28.


A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse.

Porensky PN, Mitrpant C, McGovern VL, Bevan AK, Foust KD, Kaspar BK, Wilton SD, Burghes AH.

Hum Mol Genet. 2012 Apr 1;21(7):1625-38. doi: 10.1093/hmg/ddr600. Epub 2011 Dec 20.


Recombinant AAV delivery to the central nervous system.

Bockstael O, Foust KD, Kaspar B, Tenenbaum L.

Methods Mol Biol. 2011;807:159-77. doi: 10.1007/978-1-61779-370-7_7.


Astrocytes from familial and sporadic ALS patients are toxic to motor neurons.

Haidet-Phillips AM, Hester ME, Miranda CJ, Meyer K, Braun L, Frakes A, Song S, Likhite S, Murtha MJ, Foust KD, Rao M, Eagle A, Kammesheidt A, Christensen A, Mendell JR, Burghes AH, Kaspar BK.

Nat Biotechnol. 2011 Aug 10;29(9):824-8. doi: 10.1038/nbt.1957.


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. Epub 2011 Aug 2.


A role for glia in the progression of Rett's syndrome.

Lioy DT, Garg SK, Monaghan CE, Raber J, Foust KD, Kaspar BK, Hirrlinger PG, Kirchhoff F, Bissonnette JM, Ballas N, Mandel G.

Nature. 2011 Jun 29;475(7357):497-500. doi: 10.1038/nature10214.


Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery.

Bevan AK, Hutchinson KR, Foust KD, Braun L, McGovern VL, Schmelzer L, Ward JG, Petruska JC, Lucchesi PA, Burghes AH, Kaspar BK.

Hum Mol Genet. 2010 Oct 15;19(20):3895-905. doi: 10.1093/hmg/ddq300. Epub 2010 Jul 16.


Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.

Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AH, Kaspar BK.

Nat Biotechnol. 2010 Mar;28(3):271-4. doi: 10.1038/nbt.1610. Epub 2010 Feb 28.


Over the barrier and through the blood: to CNS delivery we go.

Foust KD, Kaspar BK.

Cell Cycle. 2009 Dec 15;8(24):4017-8. Epub 2009 Dec 5. No abstract available.


AAV as a gene transfer vector for the treatment of neurological disorders: novel treatment thoughts for ALS.

Hester ME, Foust KD, Kaspar RW, Kaspar BK.

Curr Gene Ther. 2009 Oct;9(5):428-33. Review.


Nigrostriatal rAAV-mediated GDNF overexpression induces robust weight loss in a rat model of age-related obesity.

Manfredsson FP, Tumer N, Erdos B, Landa T, Broxson CS, Sullivan LF, Rising AC, Foust KD, Zhang Y, Muzyczka N, Gorbatyuk OS, Scarpace PJ, Mandel RJ.

Mol Ther. 2009 Jun;17(6):980-91. doi: 10.1038/mt.2009.45. Epub 2009 Mar 10.


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. Epub 2008 Dec 21.


Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders.

Mandel RJ, Manfredsson FP, Foust KD, Rising A, Reimsnider S, Nash K, Burger C.

Mol Ther. 2006 Mar;13(3):463-83. Epub 2006 Jan 18. Review.

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