Recent publications
Glial Dysfunction and Its Contribution to the Pathogenesis of the Neuronal Ceroid Lipofuscinoses
April 4, 2022
Takahashi K, Nelvagal HR, Lange J, Cooper JD (2022) Glial Dysfunction and Its Contribution to the Pathogenesis of the Neuronal Ceroid Lipofuscinoses. Front. Neurol. 13:886567. doi: 10.3389/fneur.2022.886567
Neuropathology of murine Sanfilippo D syndrome
November 24, 2021
Takahashi K, Le SQ, Kan S, Jansen MJ, Dickson PI, Cooper JD. Neuropathology of murine Sanfilippo D syndrome. Mol Genet Metab..2021 Dec;134(4):323-329. doi: 10.1016/j.ymgme.2021.11.010 PMID:34844863
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice
December 31, 2020
White KA, Nelvagal HR, Poole TA, Lu B, Johnson TB, Davis S, Pratt MA, Brudvig J, Assis AB, Likhite S, Meyer K, Kaspar BK, Cooper JD, Wang S, Weimer JM. Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice. Mol Ther Methods Clin Dev..2021; 20:497-507. doi: 10.1016/j.omtm.2020.12.014 PMID:33665223
Comparative proteomic profiling reveals mechanisms for early spinal cord vulnerability in CLN1 disease
September 16, 2020
Nelvagal HR, Hurtado ML, Eaton SL, Kline RA, Lamont DJ, Sands MS, Wishart TM, Cooper JD. Comparative proteomic profiling reveals mechanisms for early spinal cord vulnerability in CLN1 disease. Sci Rep.. 2020;10(1):15157. doi: 10.1038/s41598-020-72075-7 PMID: 32938982
Spinal manifestations of CLN1 disease start during the early postnatal period
September 7, 2020
Nelvagal HR, Dearborn JT, Ostergaard JR, Sands MS, Cooper JD. Spinal manifestations of CLN1 disease start during the early postnatal period. Neuropathol Appl Neurobiol.. 2020. doi: doi: 10.1111/nan.12658 PMID: 32841420
Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice
June 14, 2020
Le SQ, Nestrasil I, Kan SH, Egeland M, Cooper JD, Elashoff D, Guo R, Tolar J, Yee JK, Dickson PI. Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice. Lipids. 2020. doi:10.1002/lipd.12261 PMID: 32537944
Future perspectives: What lies ahead for Neuronal Ceroid Lipofuscinosis research?
September 1, 2020
Cooper JD, Mole SE. Future perspectives: What lies ahead for Neuronal Ceroid Lipofuscinosis research? Biochim Biophys Acta Mol Basis Dis. 2020;(1866 (9)):165681. doi:10.1016/j.bbadis.2020.165681. PMID: 31926264
Pathomechanisms in the neuronal ceroid lipofuscinoses
October 31, 2019
Nelvagal HR, Lange J, Takahashi K, Tarczyluk-Wells MA, Cooper JD. Pathomechanisms in the neuronal ceroid lipofuscinoses. Biochim Biophys Acta Mol Basis Dis.. 2019;(1866(9)):165570. doi: 10.1016/j.bbadis.2019.165570 PMID: 31678162
Combined Anti-inflammatory and Neuroprotective Treatments Have the Potential to Impact Disease Phenotypes in Cln3 -/- Mice
September 11, 2019
Tarczyluk-Wells MA, Salzlechner C, Najafi AR, Lim MJ, Smith D, Platt FM, Williams BP, Cooper JD. Combined Anti-inflammatory and Neuroprotective Treatments Have the Potential to Impact Disease Phenotypes in Cln3 -/- Mice. Frontiers in Neurology. 2019;10:963. doi:10.3389/fneur.2019.00963 PMID: 31572287
CRISPR/Cas9 mediated generation of an ovine model for infantile neuronal ceroid lipofuscinosis (CLN1 disease)
July 9, 2019
Eaton SL, Proudfoot C, Lillico SG, Skehel P, Kline RA, Hamer K, Rzechorzek NM, Clutton E, Gregson R, King T, O’Neill CA, Cooper JD, Thompson G, Whitelaw CB, Wishart TM. CRISPR/Cas9 mediated generation of an ovine model for infantile neuronal ceroid lipofuscinosis (CLN1 disease). Scientific Reports. 2019. PMID: 31289301
Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis
January 1, 2019
Mole SE, Anderson G, Band HA, Berkovic SF, Cooper JD, Kleine Holthaus SM, McKay TR, Medina DL, Rahim AA, Schulz A, Smith AJ.. Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis. Lancet neurology. 2019;18(1):107-116. PMID: 30470609
Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice
September 21, 2018
Clarke D, Pearse Y, Kan SH, Le SQ, Sanghez V, Cooper JD, Dickson PI, Iacovino M. Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice. Mol Ther Methods Clin Dev. 2018 Jul 23;10:113-127. doi: 10.1016/j.omtm.2018.06.005. PMID: 30101150; PMCID: PMC6076361.
Compromised astrocyte function and survival negatively impact neurons in infantile neuronal ceroid lipofuscinosis
August 8, 2018
Lange J, Haslett LJ, Lloyd-Evans E, Pocock JM, Sands MS, Williams BP, Cooper JD. Compromised astrocyte function and survival negatively impact neurons in infantile neuronal ceroid lipofuscinosis. Acta Neuropathol Commun. 2018 Aug 8;6(1):74. doi: 10.1186/s40478-018-0575-4. PMID: 30089511; PMCID: PMC6081811.
- Neuronal deletion of the circadian clock gene Bmal1 induces cell-autonomous dopaminergic neurodegenerationKanan, M. K., Sheehan, P. W., Haines, J. N., Gomez, P. G., Dhuler, A., Nadarajah, C. J., Wargel, Z. M., Freeberg, B. M., Nelvagal, H. R., Izumo, M., Takahashi, J. S., Cooper, J. D., Davis, A. A. & Musiek, E. S., Jan 2024, In: JCI Insight. 9, 2, e162771.Research output: Contribution to journal › Article › peer-review
- A mouse mutant deficient in both neuronal ceroid lipofuscinosis-associated proteins CLN3 and TPP1Sleat, D. E., Banach-Petrosky, W., Larrimore, K. E., Nemtsova, Y., Wiseman, J. A., Najafi, A., Johnson, D., Poole, T. A., Takahashi, K., Cooper, J. D. & Lobel, P., Jul 2023, In: Journal of Inherited Metabolic Disease. 46, 4, p. 720-734 15 p.Research output: Contribution to journal › Article › peer-review
- Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse modelTakahashi, K., Eultgen, E. M., Wang, S. H., Rensing, N. R., Nelvagal, H. R., Dearborn, J. T., Danos, O., Buss, N., Sands, M. S., Wong, M. & Cooper, J. D., Jun 15 2023, In: Journal of Clinical Investigation. 133, 12, e165908.Research output: Contribution to journal › Article › peer-review
- Severe central nervous system demyelination in Sanfilippo diseaseTaherzadeh, M., Zhang, E., Londono, I., De Leener, B., Wang, S., Cooper, J. D., Kennedy, T. E., Morales, C. R., Chen, Z., Lodygensky, G. A. & Pshezhetsky, A. V., 2023, In: Frontiers in Molecular Neuroscience. 16, 1323449.Research output: Contribution to journal › Article › peer-review
- Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the diseasePearse, Y., Clarke, D., Kan, S. H., Le, S. Q., Sanghez, V., Luzzi, A., Pham, I., Nih, L. R., Cooper, J., Dickson, P. I. & Iacovino, M., Dec 8 2022, In: Molecular Therapy – Methods and Clinical Development. 27, p. 452-463 12 p.Research output: Contribution to journal › Article › peer-review
- Effects of chronic cannabidiol in a mouse model of naturally occurring neuroinflammation, neurodegeneration, and spontaneous seizuresDearborn, J. T., Nelvagal, H. R., Rensing, N. R., Takahashi, K., Hughes, S. M., Wishart, T. M., Cooper, J. D., Wong, M. & Sands, M. S., Dec 2022, In: Scientific reports. 12, 1, 11286.Research output: Contribution to journal › Article › peer-review
- Top-down and bottom-up propagation of disease in the neuronal ceroid lipofuscinosesOstergaard, J. R., Nelvagal, H. R. & Cooper, J. D., Nov 11 2022, In: Frontiers in Neurology. 13, 1061363.Research output: Contribution to journal › Article › peer-review
- Brain volume in chronic ketamine users — relationship to sub-threshold psychotic symptoms and relevance to schizophreniaChesters, R. A., Pepper, F., Morgan, C., Cooper, J. D., Howes, O. D., Vernon, A. C. & Stone, J. M., Nov 2022, In: Psychopharmacology. 239, 11, p. 3421-3429 9 p.Research output: Contribution to journal › Article › peer-review
- Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheepNelvagal, H. R., Eaton, S. L., Wang, S. H., Eultgen, E. M., Takahashi, K., Le, S. Q., Nesbitt, R., Dearborn, J. T., Siano, N., Puhl, A. C., Dickson, P. I., Thompson, G., Murdoch, F., Brennan, P. M., Gray, M., Greenhalgh, S. N., Tennant, P., Gregson, R., Clutton, E. & Nixon, J. & 10 others, Proudfoot, C., Guido, S., Lillico, S. G., Whitelaw, C. A. B., Lu, J. Y., Hofmann, S. L., Ekins, S., Sands, M. S., Wishart, T. M. & Cooper, J. D., Oct 17 2022, In: Journal […]
- Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIBEllinwood, N. M., Valentine, B. N., Hess, A. S., Jens, J. K., Snella, E. M., Jamil, M., Hostetter, S. J., Jeffery, N. D., Smith, J. D., Millman, S. T., Parsons, R. L., Butt, M. T., Chandra, S., Egeland, M. T., Assis, A. B., Nelvagal, H. R., Cooper, J. D., Nestrasil, I., Mueller, B. A. & Labounek, R. & 17 others, Paulson, A., Prill, H., Liu, X. Y., Zhou, H., Lawrence, R., Crawford, B. E., Grover, A., Cherala, G., Melton, A. C., Cherukuri, A., Vuillemenot, B. […]