Gissen, Paul

Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy

Eichler, F., Duncan, C., Musolino, P. L., Orchard, P. J., De Oliveira, S., Thrasher, A. J., Armant, M., Dansereau, C., Lund, T. C., Miller, W. P., et al. (2017) · New England Journal of Medicine

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 604

α-synuclein oligomers interact with ATP synthase and open the permeability transition pore in Parkinson’s disease

Ludtmann, M. H. R., Angelova, P. R., Horrocks, M. H., Choi, M. L., Rodrigues, M., Baev, A. Y., Berezhnov, A. V., Yao, Z., Little, D., Banushi, B., et al. (2018) · Nature Communications

Parkinson's Disease Mechanisms and TreatmentsMitochondrial Function and Pathology

Cited by 513

Study of Intraventricular Cerliponase Alfa for CLN2 Disease

Schulz, A., Ajayi, T., Specchio, N., de Los Reyes, E., Gissen, P., Ballon, D., Dyke, J. P., Cahan, H., Slasor, P., Jacoby, D., et al. (2018) · New England Journal of Medicine

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 489

Mutations in SLC12A5 in epilepsy of infancy with migrating focal seizures

Stödberg, T., McTague, A., Ruiz, A. J., Hirata, H., Zhen, J., Long, P., Farabella, I., Meyer, E., Kawahara, A., Vassallo, G., et al. (2015) · Nature Communications

Epilepsy research and treatmentIon channel regulation and function

Cited by 184

Reproducibility of Molecular Phenotypes after Long-Term Differentiation to Human iPSC-Derived Neurons: A Multi-Site Omics Study

Volpato, V., Smith, J., Sandor, C., Ried, J. S., Baud, A., Handel, A., Newey, S. E., Wessely, F., Attar, M., Whiteley, E., et al. (2018) · Stem Cell Reports

Pluripotent Stem Cells ResearchSingle-cell and spatial transcriptomics

Cited by 165

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort

Perocheau, D. P., Cunningham, S. C., Lee, J., Antinao Diaz, J., Waddington, S. N., Gilmour, K., Eaglestone, S., Lisowski, L., Thrasher, A. J., Alexander, I. E., et al. (2019) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 81

Ophthalmic follow‐up of patients with tyrosinaemia type I on NTBC

Gissen, P., Preece, M. A., Willshaw, H. A., & McKiernan, P. J. (2003) · Journal of Inherited Metabolic Disease

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 49

De Novo Truncating Mutations in WASF1 Cause Intellectual Disability with Seizures

Ito, Y., Carss, K. J., Duarte, S. T., Hartley, T., Keren, B., Kurian, M. A., Marey, I., Charles, P., Mendonça, C., Nava, C., et al. (2018) · The American Journal of Human Genetics

Genetics and Neurodevelopmental DisordersGenomics and Rare Diseases

Cited by 41

Disorders of Tyrosine Metabolism

Chakrapani, A., Gissen, P., & McKiernan, P. (2012) · Inborn Metabolic Diseases

Metabolism and Genetic DisordersAmino Acid Enzymes and Metabolism

Cited by 17