The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse-Reference-Cited by-同舟云学术

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The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse

Published:2012-01 Issue:1 Volume:105 Page:116-125
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Wang Dan,Belakhov Valery,Kandasamy Jeyakumar,Baasov Timor,Li Su-Chen,Li Yu-Teh,Bedwell David M.,Keeling Kim M.

Funder

NIH/NINDS

University of Pennsylvania

NIH/NIGMS

US–Israel Binational Science Foundation

Center of Absorption in Science

Ministry of Immigration Absorption

Ministry of Science and Technology, Israel

Schulich

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference57 articles.

1. Suppression of nonsense mutations as a therapeutic approach to treat genetic diseases;Keeling;WIREs RNA,2011

2. Recoding therapies for genetic diseases;Keeling,2010

3. Introducing sense into nonsense in treatments of human genetic diseases;Linde;Trends Genet.,2008

4. Nonaminoglycoside compounds induce readthrough of nonsense mutations;Du;J. Exp. Med.,2009

5. PTC124 targets genetic disorders caused by nonsense mutations;Welch;Nature,2007

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3. Pharmaceuticals Promoting Premature Termination Codon Readthrough: Progress in Development;Biomolecules;2023-06-14

4. Triamterene Functions as an Effective Nonsense Suppression Agent for MPS I-H (Hurler Syndrome);International Journal of Molecular Sciences;2023-02-24

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