Correction: Reporting preclinical gene therapy studies in the field of Niemann-Pick type C disease according to the ARRIVE guidelines.
作者信息
Rasmussen Charlotte Laurfelt Munch, Burkhart Annette, Moos Torben, Thomsen Louiza Bohn
机构信息
Neurobiology Research and Drug Delivery, Department of Health Science and Technology, Aalborg University, Selma Lagerløfs Vej 249, DK-9260, Gistrup, Denmark.
The Biomedical Laboratory, Department of Molecular Medicine, University of Southern Denmark, J.B. Winsløws Vej 23, DK-5000, Odense C, Denmark.
出版信息
Orphanet J Rare Dis. 2025 Jun 10;20(1):293. doi: 10.1186/s13023-025-03826-w.
Abstract
摘要
相似文献
1
Correction: Reporting preclinical gene therapy studies in the field of Niemann-Pick type C disease according to the ARRIVE guidelines.更正：根据ARRIVE指南报告尼曼-匹克C型病领域的临床前基因治疗研究。
Orphanet J Rare Dis. 2025 Jun 10;20(1):293. doi: 10.1186/s13023-025-03826-w.
2
Reporting preclinical gene therapy studies in the field of Niemann-Pick type C disease according to the ARRIVE guidelines.根据ARRIVE指南报告尼曼-匹克C型病领域的临床前基因治疗研究。
Orphanet J Rare Dis. 2025 May 6;20(1):214. doi: 10.1186/s13023-024-03479-1.
3
Peripheral neuropathy as a very rare symptom in a patient with Niemann-Pick type C with negative enzymatic evaluation: a case report.尼曼-皮克 C 型患者外周神经病为非常罕见症状，且酶学评估为阴性：病例报告。
J Med Case Rep. 2022 Jan 12;16(1):23. doi: 10.1186/s13256-021-03136-2.
4
Visceral symptoms as a key diagnostic sign for the early infantile form of Niemann-Pick disease type C in a Russian patient: a case report.内脏症状作为俄罗斯一名患者早发型尼曼-匹克病C型的关键诊断体征：病例报告
J Med Case Rep. 2016 Jun 1;10(1):143. doi: 10.1186/s13256-016-0925-4.
5
[Three Chinese children with Niemann-Pick disease type C with neonatal cholestasis as initial presentation].[三名以新生儿胆汁淤积为首发表现的C型尼曼-匹克病中国儿童]
Zhonghua Er Ke Za Zhi. 2015 Jan;53(1):57-61.
6
Impact of miglustat on evolution of atypical presentation of late-infantile-onset Niemann-Pick disease type C with early cognitive impairment, behavioral dysfunction, epilepsy, ophthalmoplegia, and cerebellar involvement: a case report.米格鲁司他对伴有早期认知障碍、行为功能障碍、癫痫、眼肌麻痹和小脑受累的晚发性婴儿型尼曼-匹克病C型非典型表现演变的影响：一例报告
J Med Case Rep. 2016 Sep 6;10(1):241. doi: 10.1186/s13256-016-1038-9.
7
Understanding Niemann-Pick type C disease: a fat problem.了解尼曼-匹克C型病：一个脂肪问题。
Curr Opin Neurol. 2003 Apr;16(2):155-61. doi: 10.1097/01.wco.0000063764.15877.1c.
8
Molecular basis for a new bovine model of Niemann-Pick type C disease.Niemann-Pick 型 C 病新型牛模型的分子基础。
PLoS One. 2020 Sep 24;15(9):e0238697. doi: 10.1371/journal.pone.0238697. eCollection 2020.
9
A Rare Case of Niemann-Pick Disease Type-A.一例罕见的A型尼曼-匹克病病例。
Cureus. 2024 Apr 30;16(4):e59427. doi: 10.7759/cureus.59427. eCollection 2024 Apr.
10
Characterization of Niemann-Pick diseases genes mutation spectrum in Iran and identification of a novel mutation in gene.伊朗尼曼-匹克病基因突变谱特征分析及一个新突变的鉴定
Med J Islam Repub Iran. 2019 Nov 25;33:126. doi: 10.34171/mjiri.33.126. eCollection 2019.
本文引用的文献
1
Reporting preclinical gene therapy studies in the field of Niemann-Pick type C disease according to the ARRIVE guidelines.根据ARRIVE指南报告尼曼-匹克C型病领域的临床前基因治疗研究。
Orphanet J Rare Dis. 2025 May 6;20(1):214. doi: 10.1186/s13023-024-03479-1.
Zotero 插件