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Paul Gissen

university college london
H-index:62
Citation Count:1.4万
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Longitudinal outcomes of non-transplanted ornithine transcarbamylase deficiency in males and females: a multicentre UK natural history cohort

非移植鸟氨酸转氨甲酰酶缺乏症在男性和女性中的纵向结局：一项多中心英国自然病史队列研究

Orphanet Journal of Rare Diseases
IF3.5
2026-07-280
OA
Berna Seker-Yilmaz; Julien Baruteau; Raphael Buttigieg; Anupam Chakrapani; Michael Champion; Catherine Davies; James Davison; Marjorie Dixon; Sherry Fang; Emma Footitt; Joanna Gribben; Stephanie Grunewald; Gemma Hack; Thomas Hartley; Hugh Lemonde; Philippa Mills; Andrew A. M. Morris; Helen Mundy; Dinusha Pandithan; Radha Ramachandran; Siyamini Sivananthan; Chern Tan; Dexter Tarr; Louise Van Dorp; Roshni Vara; Abigail Woodward; Paul Gissen
Identification of AAV Vector Integration Sites in Primary Human and Macaque Hepatocytes in vivo, and Analysis of Oncogenic Risk

体内鉴定人源和食蟹猴原代肝细胞中AAV载体整合位点及分析致癌风险

Molecular Therapy
IF12
2026-07-220
OA
Suzanne Scott; Claus V. Hallwirth; Natsuki Sasaki; Sharon C. Cunningham; Erhua Zhu; Neeta Khandekar; Eddy Kizana; Julien Baruteau; Paul Gissen; Dany P. Perocheau; Denis C. Bauer; Laurence O.W. Wilson; Leszek Lisowski; Ian E. Alexander
Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models

在ARC综合征动物模型中体内慢病毒基因治疗的安全性与有效性分析

Nature Communications
IF15.7
2026-06-190
OAAI
Claudiu A. Cozmescu; Mina Nazari; Loukia Touramanidou; Sonam Gurung; Dany Perocheau; Neil Sebire; Yi-Ting Hu; Sian Goldsworthy; Jemima J. Burden; Youssef Khalil; Ivan Doykov; John R. Counsell; Rajvinder Karda; Sergi Castellano; Philippa Mills; Peter Clayton; Wendy Heywood; Dale Moulding; Simon N. Waddington; Julien Baruteau; Giandomenico Turchiano; Paul Gissen
A roadmap for a patient-centred approach to Pompe disease management

针对庞贝病管理的以患者为中心的方法路线图

Journal of Neurology
IF4.6
2026-02-140
OAAI
Benedikt Schoser; Cristina Domínguez-González; Pascal Laforet; Andreas Hahn; Paul Gissen; Anna Kostera-Pruszczyk; Andreas Thalmeier; John Vissing
Pilot Study of Fingolimod Treatment in Neuronal Ceroid Lipofuscinosis Type 1

苯丁酸甘油酯治疗神经元蜡样脂褐质沉积症1型的初步研究

Neurology-Genetics
IF3.7
2026-02-010
PRE
Messina, Martina; Whiteley, Rebecca; Gan, Chin; Heywood, Wendy E.; Heslegrave, Amanda J.; Keating, Megan; Taylor, Sophie; Footitt, Emma; Mills, Philippa; Hemingway, Cheryl; Gissen, Paul; Vecchiato, Katy
From Neonatal Encephalopathy to Adult Survival: Revisiting the Natural History of D-Bifunctional Protein Deficiency in a Multicentre International Case Series

从新生儿脑病到成人存活：多中心国际病例系列中重新审视D-双功能蛋白缺乏的自然史

JOURNAL OF INHERITED METABOLIC DISEASE
IF3.8
2025-12-090
PRE
Diaz-Moreno, U.; Brothwell, S. L. C.; Darling, A.; Baide-Mairena, H.; Lemonde, H.; Turon-Vinas, E.; Korenev, S.; Ibanez, S.; Beesley, C.; Gissen, P.; Parida, A.; Santra, S.; Wassmer, E.; Batzios, S.
Evidence of the impact of CLN2 and CLN3 Batten disease on families in the United Kingdom

英国家庭中CLN2和CLN3巴顿病对家庭影响的证据

ORPHANET JOURNAL OF RARE DISEASES
IF3.5
2025-05-120
OA
Mole, Sara E.; Gissen, Paul; Nordstrom, Shannon; Wait, Suzanne; Allen, Louise; Antonini, Mathilda; Brownnutt, Liz; Brown, Richard; Cole, Barbara; Gibbon, Frances; Henderson, Robert H.; Kenrick, Sarah; Sisic, Zlatko; Thompson, Bob; Nightingale, Joanna
Communication and eating & drinking skills in five children with CLN2 at the time of diagnosis
MOLECULAR GENETICS AND METABOLISM
IF3.5
2025-02-010
PRE
Pritchard, Melissa; Quinn, Sarah; Gissen, Paul
Speech and language therapy assessment of communication and eating & drinking skills in children with CLN2 presented alongside Hamburg LINCL Scale Language Scores & Weill-Cornell LINCL feeding scores
MOLECULAR GENETICS AND METABOLISM
IF3.5
2025-02-010
PRE
Pritchard, Melissa; Quinn, Sarah; Gissen, Paul
Long-term findings of N-acetyl-L-leucine for Niemann-Pick disease type C
MOLECULAR GENETICS AND METABOLISM
IF3.5
2025-02-010
PRE
Bremova-Ertl, Tatiana; Gautschi, Matthias; Gissen, Paul; Hahn, Andreas; Jones, Simon; Mengel, Eugen; Arash-Kaps, Laila; Mendoz, Grecia; Park, Julien H.; Ramaswami, Uma; Martakis, Kyriakos; Brands, Marion
Clinical Features, Biochemistry, Imaging, and Treatment Response in a Single-Center Cohort With Coenzyme Q10 Biosynthesis Disorders
NEUROLOGY-GENETICS
IF3.7
2024-12-011
PRE
Wahedi, Azizia; Sudhakar, Sniya; Lam, Amanda; Ciancio, Jose Ignacio Rodriguez; Mills, Philippa; Gissen, Paul; Gardham, Alice; Kapadia, Jogesh; Hassell, Jane; Heales, Simon; Rahman, Shamima
Macrophage Inhibitor Clodronate Enhances Liver Transduction of Lentiviral but Not Adeno-Associated Viral Vectors or mRNA Lipid Nanoparticles in Neonatal and Juvenile Mice

巨噬细胞抑制剂氯膦酸钠增强新生和幼年小鼠慢病毒而不是腺相关病毒载体或mRNA脂质纳米颗粒的肝脏转导

CELLS
IF5.2
2024-11-290
OAAI
Touramanidou, Loukia; Gurung, Sonam; Cozmescu, Claudiu A.; Perocheau, Dany; Moulding, Dale; Finn, Patrick F.; Frassetto, Andrea; Waddington, Simon N.; Gissen, Paul; Baruteau, Julien
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy

慢病毒基因治疗脑型肾上腺脑白质营养不良

NEW ENGLAND JOURNAL OF MEDICINE
IF78.5
2024-10-104
PRE
Eichler, Florian; Duncan, Christine N.; Musolino, Patricia L.; Lund, Troy C.; Gupta, Ashish O.; De Oliveira, Satiro; Thrasher, Adrian J.; Aubourg, Patrick; Kuehl, Joern-Sven; Loes, Daniel J.; Amartino, Hernan; Smith, Nicholas; Fernandes, Juliana Folloni; Sevin, Caroline; Sankar, Raman; Hussain, Shaun A.; Gissen, Paul; Dalle, Jean-Hugues; Platzbecker, Uwe; Downey, Gerald F.; McNeil, Elizabeth; Demopoulos, Laura; Dietz, Andrew C.; Thakar, Himal L.; Orchard, Paul J.; Williams, David A.
Evolution of Movement Disorders in Patients With CLN2-Batten Disease Treated With Enzyme Replacement Therapy
NEUROLOGY
IF8.5
2024-08-133
PRE
Spaull, Robert; Soo, Audrey K.; Batzios, Spyros; Footitt, Emma; Whiteley, Rebecca; Mink, Jonathan W.; Carr, Lucinda; Gissen, Paul; Kurian, Manju A.
Enzyme Replacement Therapy for CLN2 Disease: MRI Volumetry Shows Significantly Slower Volume Loss Compared with a Natural History Cohort
AMERICAN JOURNAL OF NEURORADIOLOGY
IF3.7
2024-07-082
PRE
Gaur, Pritika; Gissen, Paul; Biswas, Asthik; Mankad, Kshitij; Sudhakar, Sniya; D'Arco, Felice; Schulz, Angela; Fiehler, Jens; Sedlacik, Jan; Lobel, Ulrike
Trial of N-Acetyl-L-Leucine in Niemann-Pick Disease Type C
NEW ENGLAND JOURNAL OF MEDICINE
IF78.5
2024-02-018
PRE
Bremova-Ertl, Tatiana; Ramaswami, Uma; Brands, Marion; Foltan, Tomas; Gautschi, Matthias; Gissen, Paul; Gowing, Francesca; Hahn, Andreas; Jones, Simon; Kay, Richard; Kolnikova, Miriam; Arash-Kaps, Laila; Marquardt, Thorsten; Mengel, Eugen; Park, Julien H.; Reichmannova, Stella; Schneider, Susanne A.; Sivananthan, Siyamini; Walterfang, Mark; Wibawa, Pierre; Strupp, Michael; Martakis, Kyriakos
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
IF3.5
2024-02-010
PRE
Ohnsman, Christina; Bailey, Alexander M.; Huang, Wei; Buss, Nicholas; Kim, Kwi Hye; Chan, Gary; Burke, Jenna; Phillips, Dawn; Higgins, Mikayla; Henderson, Robert H.; Gissen, Paul; Schulz, Angela; Pakola, Stephen; Falabella, Paulo
Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children under 3 years of age
MOLECULAR GENETICS AND METABOLISM
IF3.5
2024-02-010
PRE
Schulz, Angela; Reyes, Emily C. de Los; Specchio, Nicola; Gissen, Paul; Slasor, Peter; Bondade, Shailesh; Pfeffer, Jessica L. Cohen
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
IF3.5
2024-02-010
PRE
Fields, Taylor; Bremova-Ertl, Tatiana; Ramaswami, Uma; Brands, Marion; Gautschi, Matthias; Gissen, Paul; Hahn, Andreas; Jones, Simon; Kolnikova, Miriam; Arash-Karps, Laila; Mengel, Eugen; Park, Julien; Reichmannova, Stella; Schneider, Susanne; Sivananthan, Siyamini; Walterfang, Mark; Wibawa, Pierre; Strupp, Michael; Martakis, Kyriakos
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria
SCIENCE TRANSLATIONAL MEDICINE
IF14.6
2024-01-108
OAAI
Gurung, Sonam; Timmermand, Oskar Vilhelmsson; Perocheau, Dany; Gil-Martinez, Ana Luisa; Minnion, Magdalena; Touramanidou, Loukia; Fang, Sherry; Messina, Martina; Khalil, Youssef; Spiewak, Justyna; Barber, Abigail R.; Edwards, Richard S.; Pinto, Patricia Lipari; Finn, Patrick F.; Cavedon, Alex; Siddiqui, Summar; Rice, Lisa; Martini, Paolo G. V.; Ridout, Deborah; Heywood, Wendy; Hargreaves, Ian; Heales, Simon; Mills, Philippa B.; Waddington, Simon N.; Gissen, Paul; Eaton, Simon; Ryten, Mina; Feelisch, Martin; Frassetto, Andrea; Witney, Timothy H.; Baruteau, Julien

Research Directions

The data has not been retrieved yet.
E
Eamonn R. Maher
H-index:134Papers:850Citations:6.7万
K
Kevin Mills
H-index:47Papers:203Citations:7,745
P
Philippa B. Mills
H-index:41Papers:137Citations:6,546
M
Manju A. Kurian
H-index:64Papers:304Citations:1.4万
W
Wendy Heywood
H-index:37Papers:197Citations:5,473
A
Angela Schulz
H-index:26Papers:159Citations:2,985
N
Neil V. Morgan
H-index:58Papers:197Citations:1.1万
N
Nicola Specchio
H-index:60Papers:393Citations:1.4万
M
Maureen Cleary
H-index:34Papers:152Citations:5,389
E
Emma Footitt
H-index:23Papers:75Citations:1,747