Gene therapy approaches to treat the neurodegeneration and visual failure in neuronal ceroid lipofuscinoses

Sophia Martha kleine Holthaus*, Alexander J. Smith, Sara E. Mole, Robin R. Ali

*Corresponding author for this work

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review

11 Citations (Scopus)

Abstract

Neuronal ceroid lipofuscinoses (NCLs) are a group of fatal, inherited lysosomal storage disorders mostly affecting the central nervous system of children. Symptoms include vision loss, seizures, motor deterioration and cognitive decline ultimately resulting in premature death. Studies in animal models showed that the diseases are amenable to gene supplementation therapies, and over the last decade, major advances have been made in the (pre)clinical development of these therapies. This mini-review summarises and discusses current gene therapy approaches for NCL targeting the brain and the eye.

Original languageEnglish
Title of host publicationAdvances in Experimental Medicine and Biology
PublisherSpringer New York LLC
Pages91-99
Number of pages9
DOIs
Publication statusPublished - 2018

Publication series

NameAdvances in Experimental Medicine and Biology
Volume1074
ISSN (Print)0065-2598
ISSN (Electronic)2214-8019

Keywords

  • AAV
  • Batten disease
  • Gene therapy
  • NCL
  • Neurodegeneration
  • Neuronal ceroid lipofuscinoses
  • Retinal degeneration

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