Ocular therapies for neuronal ceroid lipofuscinoses: more than meets the eye.
作者信息
Murray Samantha J, Mitchell Nadia L
机构信息
Faculty of Agricultural and Life Sciences, Lincoln University, Lincoln, New Zealand.
出版信息
Neural Regen Res. 2022 Aug;17(8):1755-1756. doi: 10.4103/1673-5374.332148.
Abstract
摘要
相似文献
1
Ocular therapies for neuronal ceroid lipofuscinoses: more than meets the eye.
Neural Regen Res. 2022 Aug;17(8):1755-1756. doi: 10.4103/1673-5374.332148.
2
Ocular Manifestations of Neuronal Ceroid Lipofuscinoses.
Semin Ophthalmol. 2021 Oct 3;36(7):582-595. doi: 10.1080/08820538.2021.1936571. Epub 2021 Jun 9.
3
Expanding the Neuroimaging Phenotype of Neuronal Ceroid Lipofuscinoses.
AJNR Am J Neuroradiol. 2020 Oct;41(10):1930-1936. doi: 10.3174/ajnr.A6726. Epub 2020 Aug 27.
4
Occurrence of and mortality from childhood neuronal ceroid lipofuscinoses in norway.
J Child Neurol. 2006 Nov;21(11):917-22. doi: 10.1177/08830738060210110801.
5
Clinical, electrophysiological, imaging, and ultrastructural description in 68 patients with neuronal ceroid lipofuscinoses and its subtypes.
Pediatr Neurol. 2014 Jan;50(1):85-95. doi: 10.1016/j.pediatrneurol.2013.08.008. Epub 2013 Oct 10.
6
Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses.
CNS Drugs. 2019 Apr;33(4):315-325. doi: 10.1007/s40263-019-00620-8.
7
Seizure susceptibility, phenotype, and resultant growth delay in the nclf and mnd mouse models of neuronal ceroid lipofuscinoses.
J Child Neurol. 2013 Sep;28(9):1137-41. doi: 10.1177/0883073813493667. Epub 2013 Jul 9.
8
Hippocampal lesions in the neuronal ceroid lipofuscinoses.
Eur J Paediatr Neurol. 2001;5 Suppl A:209-11. doi: 10.1053/eipn.2000.0464.
9
Recent studies of ovine neuronal ceroid lipofuscinoses from BARN, the Batten Animal Research Network.
Biochim Biophys Acta. 2015 Oct;1852(10 Pt B):2279-86. doi: 10.1016/j.bbadis.2015.06.013. Epub 2015 Jun 12.
10
Gene Therapy Approaches to Treat the Neurodegeneration and Visual Failure in Neuronal Ceroid Lipofuscinoses.
Adv Exp Med Biol. 2018;1074:91-99. doi: 10.1007/978-3-319-75402-4_12.
引用本文的文献
1
Genetic Reasons for Phenotypic Diversity in Neuronal Ceroid Lipofuscinoses and High-Resolution Imaging as a Marker of Retinal Disease.
Ophthalmol Sci. 2024 May 29;4(6):100560. doi: 10.1016/j.xops.2024.100560. eCollection 2024 Nov-Dec.
2
Experimental Therapeutic Approaches for the Treatment of Retinal Pathology in Neuronal Ceroid Lipofuscinoses.
Front Neurol. 2022 Apr 18;13:866983. doi: 10.3389/fneur.2022.866983. eCollection 2022.
本文引用的文献
1
Intravitreal gene therapy protects against retinal dysfunction and degeneration in sheep with CLN5 Batten disease.
Exp Eye Res. 2021 Jun;207:108600. doi: 10.1016/j.exer.2021.108600. Epub 2021 Apr 28.
2
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice.
Mol Ther Methods Clin Dev. 2021 Jan 5;20:497-507. doi: 10.1016/j.omtm.2020.12.014. eCollection 2021 Mar 12.
3
Intravitreal enzyme replacement preserves retinal structure and function in canine CLN2 neuronal ceroid lipofuscinosis.
Exp Eye Res. 2020 Aug;197:108130. doi: 10.1016/j.exer.2020.108130. Epub 2020 Jul 1.
4
Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease.
Hum Gene Ther. 2020 Jul;31(13-14):709-718. doi: 10.1089/hum.2020.038.
5
A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna.
Mol Ther. 2020 Feb 5;28(2):411-421. doi: 10.1016/j.ymthe.2019.11.012. Epub 2019 Nov 16.
6
Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease.
Mol Ther. 2018 Oct 3;26(10):2366-2378. doi: 10.1016/j.ymthe.2018.07.015. Epub 2018 Jul 17.
7
Prevention of Photoreceptor Cell Loss in a Cln6 Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells.
Mol Ther. 2018 May 2;26(5):1343-1353. doi: 10.1016/j.ymthe.2018.02.027. Epub 2018 Mar 2.
8
Intravitreal implantation of TPP1-transduced stem cells delays retinal degeneration in canine CLN2 neuronal ceroid lipofuscinosis.
Exp Eye Res. 2016 Nov;152:77-87. doi: 10.1016/j.exer.2016.09.003. Epub 2016 Sep 13.
9
AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease.
Sci Transl Med. 2015 Nov 11;7(313):313ra180. doi: 10.1126/scitranslmed.aac6191.
10
Enzyme replacement therapy delays pupillary light reflex deficits in a canine model of late infantile neuronal ceroid lipofuscinosis.
Exp Eye Res. 2014 Aug;125:164-72. doi: 10.1016/j.exer.2014.06.008. Epub 2014 Jun 19.
Zotero 插件