Browse by UCL people
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Number of items: 9.
Article
Babcock, MC;
Mikulka, CR;
Wang, B;
Chandriani, S;
Chandra, S;
Xu, Y;
Webster, K;
... Crawford, BE; + view all
(2021)
Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor.
Scientific Reports
, 11
, Article 14486. 10.1038/s41598-021-93601-1.
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Hughes, Michael Paul;
Nelvagal, Hemanth Ramesh;
Coombe-Tennant, Oliver;
Smith, Dave;
Smith, Claire;
Massaro, Giulia;
Poupon-Bejuit, Laura;
... Rahim, Ahad Abdul; + view all
(2023)
A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann–Pick Type C1 Disease.
Cells
, 12
(12)
, Article 1619. 10.3390/cells12121619.
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Massaro, Giulia;
Geard, Amy F;
Nelvagal, Hemanth R;
Gore, Katrina;
Clemo, Nadine K;
Waddington, Simon N;
Rahim, Ahad A;
(2024)
Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease.
Human Molecular Genetics
, Article ddae081. 10.1093/hmg/ddae081.
(In press).
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Nelvagal, HR;
Dearborn, JT;
Ostergaard, JR;
Sands, MS;
Cooper, JD;
(2021)
Spinal manifestations of CLN1 disease start during the early postnatal period.
Neuropathology and Applied Neurobiology
, 47
(2)
pp. 251-267.
10.1111/nan.12658.
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Nelvagal, HR;
Hurtado, ML;
Eaton, SL;
Kline, RA;
Lamont, DJ;
Sands, MS;
Wishart, TM;
(2020)
Comparative proteomic profiling reveals mechanisms for early spinal cord vulnerability in CLN1 disease.
Scientific Reports
, 10
(1)
, Article 15157. 10.1038/s41598-020-72075-7.
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Ostergaard, John R;
Nelvagal, Hemanth R;
Cooper, Jonathan D;
(2022)
Top-down and bottom-up propagation of disease in the neuronal ceroid lipofuscinoses.
Frontiers in Neurology
, 13
, Article 1061363. 10.3389/fneur.2022.1061363.
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White, KA;
Nelvagal, HR;
Poole, TA;
Lu, B;
Johnson, TB;
Davis, S;
Pratt, MA;
... Weimer, JM; + view all
(2021)
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.
Molecular Therapy - Methods and Clinical Development
, 20
pp. 497-507.
10.1016/j.omtm.2020.12.014.
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Ziółkowska, Ewa A;
Jansen, Matthew J;
Williams, Letitia L;
Wang, Sophie H;
Eultgen, Elizabeth M;
Takahashi, Keigo;
Le, Steven Q;
... Cooper, Jonathan D; + view all
(2025)
Gene therapy ameliorates bowel dysmotility and enteric neuron degeneration and extends survival in lysosomal storage disorder mouse models.
Science Translational Medicine
, 17
(781)
, Article eadj1445. 10.1126/scitranslmed.adj1445.
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Ziółkowska, Ewa A;
Wang, Sophie H;
Nelvagal, Hemanth R;
Cooper, Jonathan D;
(2026)
Immunostaining Methods for Lysosomal Storage Disorder Research.
Methods in Molecular Biology
, 2976
pp. 227-235.
10.1007/978-1-0716-4844-5_16.
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