Paul Gissen
University College London
H-index: 60
Europe-United Kingdom
Top articles of Paul Gissen
Title | Journal | Author(s) | Publication Date |
|---|---|---|---|
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria | Science translational medicine | Sonam Gurung Oskar Vilhelmsson Timmermand Dany Perocheau Ana Luisa Gil-Martinez Magdalena Minnion | 2024/1/10 |
Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children under 3 years of age | Molecular Genetics and Metabolism | Angela Schulz Emily C de los Reyes Nicola Specchio Paul Gissen Peter Slasor | 2024/2/1 |
Novel 3D Approach to Model Non-Alcoholic Fatty Liver Disease using human Pluripotent Stem Cells | bioRxiv | Carola M Morell Samantha Grace Tilson Rute A Tomaz Arash Shahsavari Andi Munteanu | 2024 |
RGX-381: Interim results from the first-in-human clinical trial of an investigational gene therapy for the treatment of ocular manifestations of CLN2 Batten disease | Molecular Genetics and Metabolism | Christina Ohnsman Alexander M Bailey Wei Huang Nicholas Buss Kwi Hye Kim | 2024/2/1 |
Liver‐directed gene therapy for inherited metabolic diseases | Julien Baruteau Nicola Brunetti‐Pierri Paul Gissen | 2024/1 | |
Trial of N-Acetyl-l-Leucine in Niemann–Pick Disease Type C | New England Journal of Medicine | Tatiana Bremova-Ertl Uma Ramaswami Marion Brands Tomas Foltan Matthias Gautschi | 2024/2/1 |
Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study | The Lancet Neurology | Angela Schulz Nicola Specchio Emily de Los Reyes Paul Gissen Miriam Nickel | 2024/1/1 |
Results of a phase III, randomized, placebo-controlled crossover trial with N-acetyl-L-leucine for Niemann-Pick disease type C | Molecular Genetics and Metabolism | Taylor Fields Tatiana Bremova-Ertl Uma Ramaswami Marion Brands Matthias Gautschi | 2024/2/1 |
Generation of induced pluripotent stem cells (UCLi024-A) from a patient with argininosuccinate lyase deficiency carrying a homozygous c. 437G> A (p. Arg146Gln) mutation | Stem Cell Research | Claire Duff Madeha Islam Onelia Gagliano Hema Pramod Hassan Rashidi | 2024/4/1 |
Fetal gene therapy | Simon N Waddington William H Peranteau Ahad A Rahim Ashley K Boyle Manju A Kurian | 2024/1 | |
Characterization of two human induced pluripotent stem cell lines derived from Batten disease patient fibroblasts harbouring CLN5 mutations | Stem Cell Research | Marisa Ofrim Daniel Little Mina Nazari Christopher J Minnis Michael J Devine | 2024/2/1 |
Treatment for lysine degradation-associated disorders | 2024/2/8 | ||
Targeting the liver to treat the eye | EMBO Molecular Medicine | Berna Seker Yilmaz Paul Gissen | 2023/4/11 |
Genetic therapy approaches for ornithine transcarbamylase deficiency | Berna Seker Yilmaz Paul Gissen | 2023/8/8 | |
Cardiac glycosides restore autophagy flux in an iPSC-derived neuronal model of WDR45 deficiency | bioRxiv | Apostolos Papandreou Nivedita Singh Lorita Gianfrancesco Dimitri Budinger Katy Barwick | 2023/9/13 |
First in man study of intravitreal tripeptidyl peptidase 1 for CLN2 retinopathy | Eye | James Wawrzynski Ana Rodriguez Martinez Dorothy Ann Thompson Dipak Ram Richard Bowman | 2023/12/4 |
Atidarsagene autotemcel for metachromatic leukodystrophy | Martina Messina Paul Gissen | 2023/2/1 | |
New perspectives in dried blood spot biomarkers for lysosomal storage diseases | International Journal of Molecular Sciences | Justyna Spiewak Ivan Doykov Apostolos Papandreou Jenny Hällqvist Philippa Mills | 2023/6/15 |
Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic … | Human Gene Therapy | Sharon C Cunningham Eva B van Dijk Erhua Zhu Maya Sugden Mawj Mandwie | 2023/9/1 |
The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria | Journal of Inherited Metabolic Disease | Sonam Gurung Saketh Karamched Dany Perocheau Kiran K Seunarine Tom Baldwin | 2023/12/4 |
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