Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1.

@article{Yoon2016ClinicalIO,
  title={Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1.},
  author={Sea Young Yoon and Jessica H. Bagel and Patricia A. O'Donnell and Charles Herman Vite and John Henry Wolfe},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2016},
  volume={24 1},
  pages={
          26-33
        }
}
Lysosomal storage diseases (LSDs) are debilitating neurometabolic disorders for most of which long-term effective therapies have not been developed. Gene therapy is a potential treatment but a critical barrier to treating the brain is the need for global correction. We tested the efficacy of cisterna magna infusion of adeno-associated virus type 1 (AAV1) expressing feline alpha-mannosidase gene in the postsymptomatic alpha-mannosidosis (AMD) cat, a homologue of the human disease. Lysosomal… CONTINUE READING
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