Experimental gene therapies for the NCLs

Wenfei Liu, Sophia Martha kleine-Holthaus, Saul Herranz-Martin, Mikel Aristorena, Sara E. Mole, Alexander J. Smith, Robin R. Ali, Ahad A. Rahim*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

12 Citations (Scopus)


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.

Original languageEnglish
Article number165772
JournalBiochimica et Biophysica Acta - Molecular Basis of Disease
Issue number9
Publication statusPublished - 1 Sept 2020


  • Batten disease
  • Clinical trials
  • Gene therapy
  • Neurodegeneration
  • Neuronal ceroid lipofuscinoses
  • Pre-clinical studies


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