Future perspectives: What lies ahead for Neuronal Ceroid Lipofuscinosis research?

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

卷:1866

Review

Cooper, Jonathan D.¹  Mole, Sara E.^2,3 

[1] Washington Univ, Sch Med, Pediat Storage Disorders Lab, Div Genet & Genom,Dept Pediat, St Louis, MO 63110 USA

[2] UCL, UCL MRC Lab Mol Cell Biol, London WC1E 6BT, England

[3] UCL, UCL Great Ormond St Inst Child Hlth, London WC1E 6BT, England

关键词: Batten disease;    Neuronal Ceroid Lipofuscinoses;    International congress;    Future perspectives;   

DOI  :  10.1016/j.bbadis.2020.165681

来源: Elsevier

【 摘 要 】

Progress is being made in all aspects of Neuronal Ceroid Lipofuscinosis (NCL) research, resulting in many recent advances. These advances encompass several areas that were previously thought intractable, ranging from basic science, through to a better understanding of the clinical presentation of different forms of NCL, therapeutic development, and new clinical trials that are underway. Increasing numbers of original NCL research papers continue to be published, and this new sense of momentum is greatly encouraging for the field. Here, we make some predictions as to what we can anticipate in the next few years.

【 授权许可】

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