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Keywords: Batten disease
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Articles
Biochem Soc Trans (2010) 38 (6): 1448–1452.
Published: 24 November 2010
...Jonathan D. Cooper The NCLs (neuronal ceroid lipofuscinoses) (also known as Batten disease) are a group of at least ten fatal inherited storage disorders. Despite the identification of many of the disease-causing genes, very little is known about the underlying disease mechanisms. However, now...
Articles
Biochem Soc Trans (2010) 38 (6): 1484–1488.
Published: 24 November 2010
... to tolerization, as well as being prohibitively expensive. Therefore, in the last two decades, there has been a lot of interest in the applicability of gene therapy for the LSDs, with a particular focus on the NCLs. Collectively, the NCLs (neuronal ceroid lipofuscinoses) (also known as Batten disease...