Experimental gene therapies for the NCLs.

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. • The neuronal ceroid lipofuscinoses are a group of rare neurodegenerative diseases that predominantly affect children. • There is an overwhelming need to develop therapies for the NCLs • Pre-clinical studies in NCL animal models have provided proof-of-concept supporting gene therapy to treat these conditions. • Clinical trials using viral vector-mediated gene therapy have been, or are being, conducted in NCL patients. [ABSTRACT FROM AUTHOR]