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Review
. 2020 Sep 1;1866(9):165772.
doi: 10.1016/j.bbadis.2020.165772. Epub 2020 Mar 24.

Experimental gene therapies for the NCLs

Wenfei Liu  1 , Sophia-Martha Kleine-Holthaus  2 , Saul Herranz-Martin  3 , Mikel Aristorena  2 , Sara E Mole  4 , Alexander J Smith  2 , Robin R Ali  5 , Ahad A Rahim  6
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Free article
Review

Experimental gene therapies for the NCLs

Wenfei Liu et al. Biochim Biophys Acta Mol Basis Dis. .
Free article

Abstract

The neuronal ceroid lipofuscinoses (NCLs), also known as Batten disease, are a group of rare monogenic neurodegenerative diseases predominantly affecting children. All NCLs are lethal and incurable and only one has an approved treatment available. To date, 13 NCL subtypes (CLN1-8, CLN10-14) have been identified, based on the particular disease-causing defective gene. The exact functions of NCL proteins and the pathological mechanisms underlying the diseases are still unclear. However, gene therapy has emerged as an attractive therapeutic strategy for this group of conditions. Here we provide a short review discussing updates on the current gene therapy studies for the NCLs.

Keywords: Batten disease; Clinical trials; Gene therapy; Neurodegeneration; Neuronal ceroid lipofuscinoses; Pre-clinical studies.

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Conflict of interest statement

Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

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