Items where authors include "Kaspar, B.K."

Article

Gomes, C., Sequeira, C., Likhite, S. et al. (7 more authors) (2022) Neurotoxic astrocytes directly converted from sporadic and familial ALS patient fibroblasts reveal signature diversities and miR-146a theragnostic potential in specific subtypes. Cells, 11 (7). 1186. ISSN 2073-4409

Iannitti, T., Scarrott, J., Likhite, S. et al. (12 more authors) (2018) Translating SOD1 gene silencing towards the clinic: A highly efficacious, off-target free and biomarker-supported strategy for familial ALS. Molecular Therapy : Nucleic Acids, 12. pp. 75-88. ISSN 2162-2531

Hautbergue, G.M. orcid.org/0000-0002-1621-261X, Castelli, L.M., Ferraiuolo, L. et al. (23 more authors) (2017) SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits. Nature Communications, 8. 16063 (2017).

Rinaldi, F., Motti, D., Ferraiuolo, L. orcid.org/0000-0001-9118-5714 et al. (1 more author) (2017) High Content Analysis in Amyotrophic Lateral Sclerosis. Molecular and Cellular Neuroscience, 80. pp. 180-191. ISSN 1044-7431

Frakes, A.E., Braun, L., Ferraiuolo, L. orcid.org/0000-0001-9118-5714 et al. (2 more authors) (2017) Additive amelioration of ALS by co-targeting independent pathogenic mechanisms. Annals of Clinical and Translational Neurology, 4 (2). pp. 76-86.

Webster, C.P., Smith, E.F., Bauer, C.S. et al. (12 more authors) (2016) The C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy. EMBO Journal, 35 (15). pp. 1656-1676.

Ferraiuolo, L. orcid.org/0000-0001-9118-5714, Meyer, K., Sherwood, T. et al. (12 more authors) (2016) Oligodendrocytes contribute to motor neuron death in ALS via SOD1 dependent mechanism. Proceedings of the National Academy of Sciences, 113 (42). E6496-E6505. ISSN 1091-6490

Sun, S., Sun, Y., Ling, S.C. et al. (11 more authors) (2015) Translational profiling identifies a cascade of damage initiated in motor neurons and spreading to glia in mutant SOD1-mediated ALS. Proceedings of the National Academy of Sciences of the United States of America (PNAS) ISSN 1091-6490, 112 (50). E6993 - E7002.

Meyer, K., Ferraiuolo, L., Schmelzer, L. et al. (11 more authors) (2014) Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: A dose-response study in mice and nonhuman primates. Molecular Therapy, 23 (3). 477 -487. ISSN 1525-0016

Proceedings Paper

Iannitti, T., Scarrot, J.M., Coldicott, I.R.P. et al. (4 more authors) (2016) Gene Therapy for Familial ALS Using AAV9 Mediated Silencing of Mutant SOD1. In: Human Gene Therapy. British Society for Gene and Cell Therapy Annual Conference, 15th April 2016, University College London Institute of Child Health. Mary Ann Liebert Inc , A12-A12.