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The sphingolipid psychosine inhibits fast axonal transport in Krabbe disease by activation of GSK3β and deregulation of molecular motors.
Cantuti Castelvetri L, Givogri MI, Hebert A, Smith B, Song Y, Kaminska A, Lopez-Rosas A, Morfini G, Pigino G, Sands M, Brady ST, Bongarzone ER. Cantuti Castelvetri L, et al. Among authors: bongarzone er. J Neurosci. 2013 Jun 12;33(24):10048-56. doi: 10.1523/JNEUROSCI.0217-13.2013. J Neurosci. 2013. PMID: 23761900 Free PMC article.
Mechanism of neuromuscular dysfunction in Krabbe disease.
Cantuti-Castelvetri L, Maravilla E, Marshall M, Tamayo T, D'auria L, Monge J, Jeffries J, Sural-Fehr T, Lopez-Rosas A, Li G, Garcia K, van Breemen R, Vite C, Garcia J, Bongarzone ER. Cantuti-Castelvetri L, et al. Among authors: bongarzone er. J Neurosci. 2015 Jan 28;35(4):1606-16. doi: 10.1523/JNEUROSCI.2431-14.2015. J Neurosci. 2015. PMID: 25632136 Free PMC article.
AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease).
Bradbury AM, Rafi MA, Bagel JH, Brisson BK, Marshall MS, Pesayco Salvador J, Jiang X, Swain GP, Prociuk ML, ODonnell PA, Fitzgerald C, Ory DS, Bongarzone ER, Shelton GD, Wenger DA, Vite CH. Bradbury AM, et al. Among authors: bongarzone er. Hum Gene Ther. 2018 Jul;29(7):785-801. doi: 10.1089/hum.2017.151. Epub 2018 Mar 14. Hum Gene Ther. 2018. PMID: 29316812 Free PMC article.
Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy.
Marshall MS, Issa Y, Jakubauskas B, Stoskute M, Elackattu V, Marshall JN, Bogue W, Nguyen D, Hauck Z, Rue E, Karumuthil-Melethil S, Zaric V, Bosland M, van Breemen RB, Givogri MI, Gray SJ, Crocker SJ, Bongarzone ER. Marshall MS, et al. Among authors: bongarzone er. Mol Ther. 2018 Mar 7;26(3):874-889. doi: 10.1016/j.ymthe.2018.01.009. Epub 2018 Jan 17. Mol Ther. 2018. PMID: 29433937 Free PMC article.
Macrophages Expressing GALC Improve Peripheral Krabbe Disease by a Mechanism Independent of Cross-Correction.
Weinstock NI, Shin D, Dhimal N, Hong X, Irons EE, Silvestri NJ, Reed CB, Nguyen D, Sampson O, Cheng YC, Lau JTY, Bongarzone ER, Kofler J, Escolar ML, Gelb MH, Wrabetz L, Feltri ML. Weinstock NI, et al. Among authors: bongarzone er. Neuron. 2020 Jul 8;107(1):65-81.e9. doi: 10.1016/j.neuron.2020.03.031. Epub 2020 May 5. Neuron. 2020. PMID: 32375064 Free PMC article.
Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.
Bradbury AM, Bagel JH, Nguyen D, Lykken EA, Pesayco Salvador J, Jiang X, Swain GP, Assenmacher CA, Hendricks IJ, Miyadera K, Hess RS, Ostrager A, ODonnell P, Sands MS, Ory DS, Shelton GD, Bongarzone ER, Gray SJ, Vite CH. Bradbury AM, et al. Among authors: bongarzone er. J Clin Invest. 2020 Sep 1;130(9):4906-4920. doi: 10.1172/JCI133953. J Clin Invest. 2020. PMID: 32773406 Free PMC article.
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