典型文献
Ocular therapies for neuronal ceroid lipofuscinoses: more than meets the eye
文献摘要：
The neuronal ceroid lipofuscinoses (NCLs), also known as Batten disease, are a group of inherited, neurodegenerative, lysosomal storage diseases typically manifesting in childhood. There are currently 13 known forms of NCL resulting from various mutations in the CLN (ceroid lipofuscinoses neuronal) genes (CLN1-8 and CLN10-14). Although varying in onset and severity, the NCLs share several phenotypic features including seizures, motor dysfunction, cognitive decline, and progressive loss of vision (Mole et al., 2011). Current treatment strategies being investigated in pre-clinical studies and early stage clinical trials primarily target the brain and spinal cord. While these potential therapeutics show promise in attenuating neurological disease, protection against retinal dysfunction and degeneration is generally ineffective or not reported.
文献关键词：
中图分类号：
作者姓名：
Samantha J.Murray;Nadia L.Mitchell
作者机构：
Faculty of Agricultural and Life Sciences,Lincoln University,Lincoln,New Zealand
文献出处：
引用格式：
[1]Samantha J.Murray;Nadia L.Mitchell-.Ocular therapies for neuronal ceroid lipofuscinoses: more than meets the eye)[J].中国神经再生研究（英文版）,2022(08):1755-1756
A类：
ceroid,lipofuscinoses,NCLs,Batten,CLN1,CLN10
B类：
Ocular,therapies,neuronal,more,than,meets,eye,also,known,group,inherited,neurodegenerative,lysosomal,storage,diseases,typically,manifesting,childhood,There,currently,forms,resulting,from,various,mutations,genes,Although,varying,onset,severity,share,several,phenotypic,features,including,seizures,motor,dysfunction,cognitive,decline,progressive,loss,vision,Mole,Current,treatment,strategies,being,investigated,pre,clinical,studies,early,stage,trials,primarily,target,brain,spinal,cord,While,these,potential,therapeutics,show,promise,attenuating,neurological,protection,against,retinal,degeneration,generally,ineffective,reported
AB值：
0.622667
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机标中图分类号，由域田数据科技根据网络公开资料自动分析生成，仅供学习研究参考。
