Chapter title |
Gene Therapy Approaches to Treat the Neurodegeneration and Visual Failure in Neuronal Ceroid Lipofuscinoses
|
---|---|
Chapter number | 12 |
Book title |
Retinal Degenerative Diseases
|
Published in |
Advances in experimental medicine and biology, January 2018
|
DOI | 10.1007/978-3-319-75402-4_12 |
Pubmed ID | |
Book ISBNs |
978-3-31-975401-7, 978-3-31-975402-4
|
Authors |
Sophia-Martha kleine Holthaus, Alexander J. Smith, Sara E. Mole, Robin R. Ali, kleine Holthaus, Sophia-Martha, Smith, Alexander J., Mole, Sara E., Ali, Robin R. |
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. |
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Unknown | 1 | 50% |
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Members of the public | 1 | 50% |
Scientists | 1 | 50% |
Mendeley readers
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Pharmacology, Toxicology and Pharmaceutical Science | 1 | 6% |
Other | 1 | 6% |
Unknown | 6 | 33% |