Lysosomal storage diseases

By F. Platt, A. d’Azzo, B. Davidson, E. Neufeld, C. Tifft. Nature Reviews Disease Primers, 2018

646 citations recorded by the source corpus.

Subfields: Cell Biology, Epidemiology, Physiology

View Paper: https://www.semanticscholar.org/paper/497aecfc5b2076e3b455d5999158eafafc6cd238

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1. J. Mendell
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3. L. Rodino-Klapac
4. Kimberly Goodspeed
5. S. Gray
6. Stephanie M Salabarria
7. B. Byrne
8. J. Tremblay

Citations in the corpus

• Current Clinical Applications of In Vivo Gene Therapy with AAVs (2020, background): “Collectively, LSDs are relatively common disorders (1:5,000 to 1:5,500), although individually rare.229–231 LSDs affect multiple organ systems but have one defining system predominating, such as hypertrophic cardiomyopathy in patients with infantile Pompe disease, CNS deficits in children with CNL2,”