Peer-reviewed veterinary case report
Cats with Sandhoff disease live longer after brain gene therapy
By Bradbury, Allison M et al.·Published in Molecular therapy : the journal of the American Society of Gene Therapy·2013·College of Veterinary Medicine, United States·View original on PubMed →
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Original publication title: Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.
- Species:
- cat
Plain-English summary
Two cats with Sandhoff disease, a serious genetic condition affecting the nervous system, were treated with a special gene therapy designed for their species. While untreated cats typically live around 4.5 months, those given the therapy lived significantly longer, with one reaching 10.4 months. The treatment involved injecting a vector that expressed a feline version of the enzyme they were missing, which helped improve their condition. This study suggests that using species-specific treatments can be more effective for certain diseases in pets.
People also search for: cat Sandhoff disease treatment · gene therapy for cats · feline genetic disorders · how long do cats live with Sandhoff disease
Abstract
Salutary responses to adeno-associated viral (AAV) gene therapy have been reported in the mouse model of Sandhoff disease (SD), a neurodegenerative lysosomal storage disease caused by deficiency of β-N-acetylhexosaminidase (Hex). While untreated mice reach the humane endpoint by 4.1 months of age, mice treated by a single intracranial injection of vectors expressing human hexosaminidase may live a normal life span of 2 years. When treated with the same therapeutic vectors used in mice, two cats with SD lived to 7.0 and 8.2 months of age, compared with an untreated life span of 4.5 ± 0.5 months (n = 11). Because a pronounced humoral immune response to both the AAV1 vectors and human hexosaminidase was documented, feline cDNAs for the hexosaminidase α- and β-subunits were cloned into AAVrh8 vectors. Cats treated with vectors expressing feline hexosaminidase produced enzymatic activity >75-fold normal at the brain injection site with little evidence of an immune infiltrate. Affected cats treated with feline-specific vectors by bilateral injection of the thalamus lived to 10.4 ± 3.7 months of age (n = 3), or 2.3 times as long as untreated cats. These studies support the therapeutic potential of AAV vectors for SD and underscore the importance of species-specific cDNAs for translational research.
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Search related cases →Original publication on PubMed: https://pubmed.ncbi.nlm.nih.gov/23689599/