Liu, W;
Kleine-Holthaus, S-M;
Herranz-Martin, S;
Aristorena, M;
Mole, SE;
Smith, AJ;
Ali, RR;
(2020)
Experimental gene therapies for the NCLs.
Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease
, 1866
(9)
, Article 165772. 10.1016/j.bbadis.2020.165772.
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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.
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