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The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis
Brasell EJ,
Chu LL,
Akpa MM,
Eshkar-Oren I,
Alroy I,
et al.
(2019)
The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis.
PLOS ONE 14(12): e0223954.
https://doi.org/10.1371/journal.pone.0223954