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The novel aminoglycoside, ELX-02, permits CTNSW138X translational read-through and restores lysosomal cystine efflux in cystinosis.
Brasell, Emma J; Chu, Lee Lee; Akpa, Murielle M; Eshkar-Oren, Idit; Alroy, Iris; Corsini, Rachel; Gilfix, Brian M; Yamanaka, Yojiro; Huertas, Pedro; Goodyer, Paul.
Affiliation
  • Brasell EJ; McGill University, Department of Human Genetics, Montreal, Canada.
  • Chu LL; Research Institute of the McGill University Health Centre, Montreal, Canada.
  • Akpa MM; Research Institute of the McGill University Health Centre, Montreal, Canada.
  • Eshkar-Oren I; McGill University, Department of Experimental Medicine, Montreal, Canada.
  • Alroy I; McGill University, Department of Experimental Medicine, Montreal, Canada.
  • Corsini R; Research Institute of the McGill University Health Centre, Montreal, Canada.
  • Gilfix BM; Research Institute of the McGill University Health Centre, Montreal, Canada.
  • Yamanaka Y; McGill University, Department of Human Genetics, Montreal, Canada.
  • Huertas P; McGill University, Department of Experimental Medicine, Montreal, Canada.
  • Goodyer P; McGill University, Department of Human Genetics, Montreal, Canada.
PLoS One ; 14(12): e0223954, 2019.
Article in En | MEDLINE | ID: mdl-31800572
Subject(s)
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Cystine / Cystinosis / Amino Acid Transport Systems, Neutral / Aminoglycosides / Lysosomes / Mutation Limits: Animals Language: En Journal: PLoS One Journal subject: CIENCIA / MEDICINA Year: 2019 Document type: Article Affiliation country: Canada Country of publication: United States
Fulltext
Add to My VHL
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XML
PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Cystine / Cystinosis / Amino Acid Transport Systems, Neutral / Aminoglycosides / Lysosomes / Mutation Limits: Animals Language: En Journal: PLoS One Journal subject: CIENCIA / MEDICINA Year: 2019 Document type: Article Affiliation country: Canada Country of publication: United States