Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis
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Title
Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis
Authors
Keywords
Batten disease, Translational research, Palmitoyl-Protein Thioesterase 1, Tripeptidyl peptidase 1, Gene therapy, RNA modulating therapies, Antisense oligonucleotides, Enzyme replacement therapy, Stem cell therapy, Lysosomal modulators, Autophagy modulators
Journal
Orphanet Journal of Rare Diseases
Volume 11, Issue 1, Pages -
Publisher
Springer Nature
Online
2016-04-16
DOI
10.1186/s13023-016-0414-2
References
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