Retrovirus-mediated Gene Therapy For Farber Disease
Farber disease is a rare lysosomal storage disease (LSD) caused by a deficiency of acid ceramidase (AC). Patients show a classic triad of symptoms including subcutaneous granulomas, laryngeal involvement and painful swollen joints. The most common and severe form has neurological manifestations and...
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| Format: | Others |
| Language: | en_ca |
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2008
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| Online Access: | http://hdl.handle.net/1807/11249 |