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Items: 1 to 50 of 93

1.

AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort.

Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, Mendell JR.

J Neuromuscul Dis. 2019;6(3):307-317. doi: 10.3233/JND-190403.

PMID:
31381526
2.

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.

Cain JT, Likhite S, White KA, Timm DJ, Davis SS, Johnson TB, Dennys-Rivers CN, Rinaldi F, Motti D, Corcoran S, Morales P, Pierson C, Hughes SM, Lee SY, Kaspar BK, Meyer K, Weimer JM.

Mol Ther. 2019 Jul 10. pii: S1525-0016(19)30311-9. doi: 10.1016/j.ymthe.2019.06.015. [Epub ahead of print]

3.

Ibuprofen enhances synaptic function and neural progenitors proliferation markers and improves neuropathology and motor coordination in Machado-Joseph disease models.

Mendonça LS, Nóbrega C, Tavino S, Brinkhaus M, Matos C, Tomé S, Moreira R, Henriques D, Kaspar BK, de Almeida LP.

Hum Mol Genet. 2019 May 25. pii: ddz097. doi: 10.1093/hmg/ddz097. [Epub ahead of print]

PMID:
31127937
4.

MRI-Guided Focused Ultrasound for Targeted Delivery of rAAV to the Brain.

Noroozian Z, Xhima K, Huang Y, Kaspar BK, Kügler S, Hynynen K, Aubert I.

Methods Mol Biol. 2019;1950:177-197. doi: 10.1007/978-1-4939-9139-6_10.

5.

Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS.

Iannitti T, Scarrott JM, Likhite S, Coldicott IRP, Lewis KE, Heath PR, Higginbottom A, Myszczynska MA, Milo M, Hautbergue GM, Meyer K, Kaspar BK, Ferraiuolo L, Shaw PJ, Azzouz M.

Mol Ther Nucleic Acids. 2018 Sep 7;12:75-88. doi: 10.1016/j.omtn.2018.04.015. Epub 2018 May 3.

6.

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.

7.

HSPB1 mutations causing hereditary neuropathy in humans disrupt non-cell autonomous protection of motor neurons.

Heilman PL, Song S, Miranda CJ, Meyer K, Srivastava AK, Knapp A, Wier CG, Kaspar BK, Kolb SJ.

Exp Neurol. 2017 Nov;297:101-109. doi: 10.1016/j.expneurol.2017.08.002. Epub 2017 Aug 7.

8.

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.

9.

SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits.

Hautbergue GM, Castelli LM, Ferraiuolo L, Sanchez-Martinez A, Cooper-Knock J, Higginbottom A, Lin YH, Bauer CS, Dodd JE, Myszczynska MA, Alam SM, Garneret P, Chandran JS, Karyka E, Stopford MJ, Smith EF, Kirby J, Meyer K, Kaspar BK, Isaacs AM, El-Khamisy SF, De Vos KJ, Ning K, Azzouz M, Whitworth AJ, Shaw PJ.

Nat Commun. 2017 Jul 5;8:16063. doi: 10.1038/ncomms16063.

10.

NEUROD1 Instructs Neuronal Conversion in Non-Reactive Astrocytes.

Brulet R, Matsuda T, Zhang L, Miranda C, Giacca M, Kaspar BK, Nakashima K, Hsieh J.

Stem Cell Reports. 2017 Jun 6;8(6):1506-1515. doi: 10.1016/j.stemcr.2017.04.013. Epub 2017 May 11.

11.

Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.

Mendell JR, Sahenk Z, Al-Zaidy S, Rodino-Klapac LR, Lowes LP, Alfano LN, Berry K, Miller N, Yalvac M, Dvorchik I, Moore-Clingenpeel M, Flanigan KM, Church K, Shontz K, Curry C, Lewis S, McColly M, Hogan MJ, Kaspar BK.

Mol Ther. 2017 Apr 5;25(4):870-879. doi: 10.1016/j.ymthe.2017.02.015. Epub 2017 Mar 6.

12.

Additive amelioration of ALS by co-targeting independent pathogenic mechanisms.

Frakes AE, Braun L, Ferraiuolo L, Guttridge DC, Kaspar BK.

Ann Clin Transl Neurol. 2017 Jan 11;4(2):76-86. doi: 10.1002/acn3.375. eCollection 2017 Feb.

13.

High content analysis in amyotrophic lateral sclerosis.

Rinaldi F, Motti D, Ferraiuolo L, Kaspar BK.

Mol Cell Neurosci. 2017 Apr;80:180-191. doi: 10.1016/j.mcn.2016.12.001. Epub 2016 Dec 11. Review.

14.

Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism.

Ferraiuolo L, Meyer K, Sherwood TW, Vick J, Likhite S, Frakes A, Miranda CJ, Braun L, Heath PR, Pineda R, Beattie CE, Shaw PJ, Askwith CC, McTigue D, Kaspar BK.

Proc Natl Acad Sci U S A. 2016 Oct 18;113(42):E6496-E6505. Epub 2016 Sep 29.

15.

Potent spinal parenchymal AAV9-mediated gene delivery by subpial injection in adult rats and pigs.

Miyanohara A, Kamizato K, Juhas S, Juhasova J, Navarro M, Marsala S, Lukacova N, Hruska-Plochan M, Curtis E, Gabel B, Ciacci J, Ahrens ET, Kaspar BK, Cleveland D, Marsala M.

Mol Ther Methods Clin Dev. 2016 Jul 13;3:16046. doi: 10.1038/mtm.2016.46. eCollection 2016.

16.

The C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy.

Webster CP, Smith EF, Bauer CS, Moller A, Hautbergue GM, Ferraiuolo L, Myszczynska MA, Higginbottom A, Walsh MJ, Whitworth AJ, Kaspar BK, Meyer K, Shaw PJ, Grierson AJ, De Vos KJ.

EMBO J. 2016 Aug 1;35(15):1656-76. doi: 10.15252/embj.201694401. Epub 2016 Jun 22.

17.

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.

18.

Glia-neuron interactions in neurological diseases: Testing non-cell autonomy in a dish.

Meyer K, Kaspar BK.

Brain Res. 2017 Feb 1;1656:27-39. doi: 10.1016/j.brainres.2015.12.051. Epub 2016 Jan 9. Review.

19.

An NF-κB--EphrinA5-Dependent Communication between NG2(+) Interstitial Cells and Myoblasts Promotes Muscle Growth in Neonates.

Gu JM, Wang DJ, Peterson JM, Shintaku J, Liyanarachchi S, Coppola V, Frakes AE, Kaspar BK, Cornelison DD, Guttridge DC.

Dev Cell. 2016 Jan 25;36(2):215-24. doi: 10.1016/j.devcel.2015.12.018. Epub 2016 Jan 14.

20.

Translational profiling identifies a cascade of damage initiated in motor neurons and spreading to glia in mutant SOD1-mediated ALS.

Sun S, Sun Y, Ling SC, Ferraiuolo L, McAlonis-Downes M, Zou Y, Drenner K, Wang Y, Ditsworth D, Tokunaga S, Kopelevich A, Kaspar BK, Lagier-Tourenne C, Cleveland DW.

Proc Natl Acad Sci U S A. 2015 Dec 15;112(50):E6993-7002. doi: 10.1073/pnas.1520639112. Epub 2015 Nov 30.

21.

Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model.

Nizzardo M, Simone C, Rizzo F, Salani S, Dametti S, Rinchetti P, Del Bo R, Foust K, Kaspar BK, Bresolin N, Comi GP, Corti S.

Sci Adv. 2015 Mar 13;1(2):e1500078. doi: 10.1126/sciadv.1500078. eCollection 2015 Mar.

22.

Macrophage migration inhibitory factor as a chaperone inhibiting accumulation of misfolded SOD1.

Israelson A, Ditsworth D, Sun S, Song S, Liang J, Hruska-Plochan M, McAlonis-Downes M, Abu-Hamad S, Zoltsman G, Shani T, Maldonado M, Bui A, Navarro M, Zhou H, Marsala M, Kaspar BK, Da Cruz S, Cleveland DW.

Neuron. 2015 Apr 8;86(1):218-32. doi: 10.1016/j.neuron.2015.02.034. Epub 2015 Mar 19.

23.

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.

24.

Perspectives on best practices for gene therapy programs.

Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, McDonald CL, McLaughlin J, Weiss McLeod B, Mendell JR, Nuckolls G, Stedman HH, Tagle DA, Vandenberghe LH, Wang H, Wernett PJ, Wilson JM, Porter JD, Gubitz AK.

Hum Gene Ther. 2015 Mar;26(3):127-33. doi: 10.1089/hum.2014.147. Epub 2015 Mar 3.

25.

Chronic oligodendrogenesis and remyelination after spinal cord injury in mice and rats.

Hesp ZC, Goldstein EZ, Miranda CJ, Kaspar BK, McTigue DM.

J Neurosci. 2015 Jan 21;35(3):1274-90. doi: 10.1523/JNEUROSCI.2568-14.2015. Erratum in: J Neurosci. 2015 May 6;35(18):7313. Goldstein, Evan A [Corrected to Goldstein, Evan Z] and Kaspar, Brain K [Corrected to Kaspar, Brian K].

26.

Transplantation of cerebellar neural stem cells improves motor coordination and neuropathology in Machado-Joseph disease mice.

Mendonça LS, Nóbrega C, Hirai H, Kaspar BK, Pereira de Almeida L.

Brain. 2015 Feb;138(Pt 2):320-35. doi: 10.1093/brain/awu352. Epub 2014 Dec 19.

PMID:
25527827
27.

A large animal model of spinal muscular atrophy and correction of phenotype.

Duque SI, Arnold WD, Odermatt P, Li X, Porensky PN, Schmelzer L, Meyer K, Kolb SJ, Schümperli D, Kaspar BK, Burghes AH.

Ann Neurol. 2015 Mar;77(3):399-414. doi: 10.1002/ana.24332. Epub 2015 Feb 9.

28.

Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex.

Thomsen GM, Gowing G, Latter J, Chen M, Vit JP, Staggenborg K, Avalos P, Alkaslasi M, Ferraiuolo L, Likhite S, Kaspar BK, Svendsen CN.

J Neurosci. 2014 Nov 19;34(47):15587-600. doi: 10.1523/JNEUROSCI.2037-14.2014.

29.

Intravenous injections in neonatal mice.

Gombash Lampe SE, Kaspar BK, Foust KD.

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

30.

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.

31.

A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.

Mendell JR, Sahenk Z, Malik V, Gomez AM, Flanigan KM, Lowes LP, Alfano LN, Berry K, Meadows E, Lewis S, Braun L, Shontz K, Rouhana M, Clark KR, Rosales XQ, Al-Zaidy S, Govoni A, Rodino-Klapac LR, Hogan MJ, Kaspar BK.

Mol Ther. 2015 Jan;23(1):192-201. doi: 10.1038/mt.2014.200. Epub 2014 Oct 17.

32.

Making sense of pain: are pluripotent stem cell-derived sensory neurons a new tool for studying pain mechanisms?

Meyer K, Kaspar BK.

Mol Ther. 2014 Aug;22(8):1403-1405. doi: 10.1038/mt.2014.123. No abstract available.

33.

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.

34.

Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept.

Arnold WD, Porensky PN, McGovern VL, Iyer CC, Duque S, Li X, Meyer K, Schmelzer L, Kaspar BK, Kolb SJ, Kissel JT, Burghes AH.

Ann Clin Transl Neurol. 2014 Jan 1;1(1):34-44.

35.

Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS.

Meyer K, Ferraiuolo L, Miranda CJ, Likhite S, McElroy S, Renusch S, Ditsworth D, Lagier-Tourenne C, Smith RA, Ravits J, Burghes AH, Shaw PJ, Cleveland DW, Kolb SJ, Kaspar BK.

Proc Natl Acad Sci U S A. 2014 Jan 14;111(2):829-32. doi: 10.1073/pnas.1314085111. Epub 2013 Dec 30.

36.

AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy.

Sahenk Z, Galloway G, Clark KR, Malik V, Rodino-Klapac LR, Kaspar BK, Chen L, Braganza C, Montgomery C, Mendell JR.

Mol Ther. 2014 Mar;22(3):511-521. doi: 10.1038/mt.2013.250. Epub 2013 Oct 28.

37.

Active and passive immunization strategies based on the SDPM1 peptide demonstrate pre-clinical efficacy in the APPswePSEN1dE9 mouse model for Alzheimer's disease.

Camboni M, Wang CM, Miranda C, Yoon JH, Xu R, Zygmunt D, Kaspar BK, Martin PT.

Neurobiol Dis. 2014 Feb;62:31-43. doi: 10.1016/j.nbd.2013.09.001. Epub 2013 Sep 8.

38.

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.

39.

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.

40.

Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model.

Rodino-Klapac LR, Janssen PM, Shontz KM, Canan B, Montgomery CL, Griffin D, Heller K, Schmelzer L, Handy C, Clark KR, Sahenk Z, Mendell JR, Kaspar BK.

Hum Mol Genet. 2013 Dec 15;22(24):4929-37. doi: 10.1093/hmg/ddt342. Epub 2013 Jul 17.

41.

Neural stem cells as a therapeutic approach for amyotrophic lateral sclerosis.

Ferraiuolo L, Frakes A, Kaspar BK.

Mol Ther. 2013 Mar;21(3):503-5. doi: 10.1038/mt.2013.24. No abstract available.

42.

Transplantation of gene-corrected motor neurons as a therapeutic strategy for spinal muscular atrophy.

Meyer K, Miranda CJ, Kaspar BK.

Mol Ther. 2013 Mar;21(3):502-3. doi: 10.1038/mt.2013.23. No abstract available.

43.

Gene delivery improvement for treating the lysosomal storage disorder metachromatic leukodystrophy.

Ferraiuolo L, Kaspar BK.

Hum Gene Ther. 2012 Aug;23(8):793-5. doi: 10.1089/hum.2012.2516. No abstract available.

PMID:
22900577
44.

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.

45.

Gene therapy for muscular dystrophy: lessons learned and path forward.

Mendell JR, Rodino-Klapac L, Sahenk Z, Malik V, Kaspar BK, Walker CM, Clark KR.

Neurosci Lett. 2012 Oct 11;527(2):90-9. doi: 10.1016/j.neulet.2012.04.078. Epub 2012 May 17. Review.

46.

Aging brain microenvironment decreases hippocampal neurogenesis through Wnt-mediated survivin signaling.

Miranda CJ, Braun L, Jiang Y, Hester ME, Zhang L, Riolo M, Wang H, Rao M, Altura RA, Kaspar BK.

Aging Cell. 2012 Jun;11(3):542-52. doi: 10.1111/j.1474-9726.2012.00816.x. Epub 2012 Apr 4.

47.

Knee extensor strength exhibits potential to predict function in sporadic inclusion-body myositis.

Lowes LP, Alfano L, Viollet L, Rosales XQ, Sahenk Z, Kaspar BK, Clark KR, Flanigan KM, Mendell JR, McDermott MP.

Muscle Nerve. 2012 Feb;45(2):163-8. doi: 10.1002/mus.22321.

48.

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.

49.

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.

50.

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.

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