logo
科言猫学术研究的AI总结
language
手机端手机端
  • Phone Feature微信小程序
简体中文简体中文
notice消息通知
博客博客
delete
arrow
返回
P

Paul Gissen

Great Ormond Street Institute of Child Health

62H指数
378论文数
1.4W被引数
收录论文 147
发表时间
logo
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
errOrphanet Journal of Rare Diseases
IF3.5
err2026-07-28
err0
errOAAI
errBerna 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
err分享
err收藏
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
errMolecular Therapy
IF12
err2026-07-22
err0
errOAAI
errSuzanne 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
err分享
err收藏
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
errNature Communications
IF15.7
err2026-06-19
err0
errOAAI
errClaudiu 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
err分享
err收藏
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
errJournal of Neurology
IF4.6
err2026-02-14
err0
errOAAI
errBenedikt Schoser; Cristina Domínguez-González; Pascal Laforet; Andreas Hahn; Paul Gissen; Anna Kostera-Pruszczyk; Andreas Thalmeier; John Vissing
err分享
err收藏
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
errNeurology-Genetics
IF3.7
err2026-02-01
err0
PREAI
errMessina, 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
err分享
err收藏
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
errJOURNAL OF INHERITED METABOLIC DISEASE
IF3.8
err2025-12-09
err0
PREAI
errDiaz-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.
err分享
err收藏
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
errORPHANET JOURNAL OF RARE DISEASES
IF3.5
err2025-05-12
err0
errOAAI
errMole, 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
err分享
err收藏
Communication and eating & drinking skills in five children with CLN2 at the time of diagnosis
errMOLECULAR GENETICS AND METABOLISM
IF3.5
err2025-02-01
err0
PREAI
errPritchard, Melissa; Quinn, Sarah; Gissen, Paul
err分享
err收藏
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
errMOLECULAR GENETICS AND METABOLISM
IF3.5
err2025-02-01
err0
PREAI
errPritchard, Melissa; Quinn, Sarah; Gissen, Paul
err分享
err收藏
Long-term findings of N-acetyl-L-leucine for Niemann-Pick disease type C
errMOLECULAR GENETICS AND METABOLISM
IF3.5
err2025-02-01
err0
PREAI
errBremova-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
err分享
err收藏
Clinical Features, Biochemistry, Imaging, and Treatment Response in a Single-Center Cohort With Coenzyme Q10 Biosynthesis Disorders
errNEUROLOGY-GENETICS
IF3.7
err2024-12-01
err1
PREAI
errWahedi, Azizia; Sudhakar, Sniya; Lam, Amanda; Ciancio, Jose Ignacio Rodriguez; Mills, Philippa; Gissen, Paul; Gardham, Alice; Kapadia, Jogesh; Hassell, Jane; Heales, Simon; Rahman, Shamima
err分享
err收藏
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
errCELLS
IF5.2
err2024-11-29
err0
errOAAI
errTouramanidou, Loukia; Gurung, Sonam; Cozmescu, Claudiu A.; Perocheau, Dany; Moulding, Dale; Finn, Patrick F.; Frassetto, Andrea; Waddington, Simon N.; Gissen, Paul; Baruteau, Julien
err分享
err收藏
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
errNEW ENGLAND JOURNAL OF MEDICINE
IF78.5
err2024-10-10
err4
PREAI
errEichler, 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.
err分享
err收藏
Evolution of Movement Disorders in Patients With CLN2-Batten Disease Treated With Enzyme Replacement Therapy
errNEUROLOGY
IF8.5
err2024-08-13
err3
PREAI
errSpaull, Robert; Soo, Audrey K.; Batzios, Spyros; Footitt, Emma; Whiteley, Rebecca; Mink, Jonathan W.; Carr, Lucinda; Gissen, Paul; Kurian, Manju A.
err分享
err收藏
Enzyme Replacement Therapy for CLN2 Disease: MRI Volumetry Shows Significantly Slower Volume Loss Compared with a Natural History Cohort
errAMERICAN JOURNAL OF NEURORADIOLOGY
IF3.7
err2024-07-08
err2
PREAI
errGaur, Pritika; Gissen, Paul; Biswas, Asthik; Mankad, Kshitij; Sudhakar, Sniya; D'Arco, Felice; Schulz, Angela; Fiehler, Jens; Sedlacik, Jan; Lobel, Ulrike
err分享
err收藏
Trial of N-Acetyl-L-Leucine in Niemann-Pick Disease Type C
errNEW ENGLAND JOURNAL OF MEDICINE
IF78.5
err2024-02-01
err8
PREAI
errBremova-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
err分享
err收藏
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
errMOLECULAR GENETICS AND METABOLISM
IF3.5
err2024-02-01
err0
PREAI
errOhnsman, 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
err分享
err收藏
Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children under 3 years of age
errMOLECULAR GENETICS AND METABOLISM
IF3.5
err2024-02-01
err0
PREAI
errSchulz, Angela; Reyes, Emily C. de Los; Specchio, Nicola; Gissen, Paul; Slasor, Peter; Bondade, Shailesh; Pfeffer, Jessica L. Cohen
err分享
err收藏
Results of a phase III, randomized, placebo-controlled crossover trial with N-acetyl-L-leucine for Niemann-Pick disease type C
errMOLECULAR GENETICS AND METABOLISM
IF3.5
err2024-02-01
err0
PREAI
errFields, 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
err分享
err收藏
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria
errSCIENCE TRANSLATIONAL MEDICINE
IF14.6
err2024-01-10
err8
errOAAI
errGurung, 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
err分享
err收藏

研究方向

暂时未获取到该数据
合作学者
合作期刊
W
William J. McKenna
H 指数: 162 · 论文数: 2.4K
M
M. Williams
H 指数: 154 · 论文数: 3.4K
S
Samuel F. Berkovic
H 指数: 145 · 论文数: 1.2K
H
Hugh Watkins
H 指数: 144 · 论文数: 914
I
Ingrid E. Scheffer
H 指数: 141 · 论文数: 1.0K
查看更多