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The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential.
Friesen WJ, Johnson B, Sierra J, Zhuo J, Vazirani P, Xue X, Tomizawa Y, Baiazitov R, Morrill C, Ren H, Babu S, Moon YC, Branstrom A, Mollin A, Hedrick J, Sheedy J, Elfring G, Weetall M, Colacino JM, Welch EM, Peltz SW. Friesen WJ, et al. Among authors: peltz sw. PLoS One. 2018 Oct 25;13(10):e0206158. doi: 10.1371/journal.pone.0206158. eCollection 2018. PLoS One. 2018. PMID: 30359426 Free PMC article.
The known oto- and nephrotoxicity associated with aminoglycosides preclude long-term use as readthrough agents. Thus, we evaluated the components of the gentamicin complex as well as the so-called "designer" aminoglycoside, NB124, for in vitro and in vivo safety. ...
The known oto- and nephrotoxicity associated with aminoglycosides preclude long-term use as readthrough agents. Thus, we evaluated th …
Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophy.
Finkel RS, Flanigan KM, Wong B, Bönnemann C, Sampson J, Sweeney HL, Reha A, Northcutt VJ, Elfring G, Barth J, Peltz SW. Finkel RS, et al. Among authors: peltz sw. PLoS One. 2013 Dec 11;8(12):e81302. doi: 10.1371/journal.pone.0081302. eCollection 2013. PLoS One. 2013. PMID: 24349052 Free PMC article. Clinical Trial.
INTERPRETATION: Ataluren showed activity and safety in this short-term study, supporting evaluation of ataluren 10, 10, 20 mg/kg and 20, 20, 40 mg/kg in a Phase 2b, double-blind, long-term study in nonsense mutation DMD. ...
INTERPRETATION: Ataluren showed activity and safety in this short-term study, supporting evaluation of ataluren 10, 10, 20 mg/kg and …
Characterization of cis-acting sequences and decay intermediates involved in nonsense-mediated mRNA turnover.
Hagan KW, Ruiz-Echevarria MJ, Quan Y, Peltz SW. Hagan KW, et al. Among authors: peltz sw. Mol Cell Biol. 1995 Feb;15(2):809-23. doi: 10.1128/MCB.15.2.809. Mol Cell Biol. 1995. PMID: 7823948 Free PMC article.
One clear example of this relationship is the observation that nonsense mutations can accelerate the decay of mRNAs in a process that we term nonsense-mediated mRNA decay. The experiments described here demonstrate that in the yeast Saccharomyces cerevisiae premature trans …
One clear example of this relationship is the observation that nonsense mutations can accelerate the decay of mRNAs in a process that we …