A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin.

A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin. Research Abstract Details 

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A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin. Abstract Text:

Chunmei Yang,Jinong Feng,Wenjia Song,Jicheng Wang,Becky Tsai,Yunwu Zhang,William A Scaringe,Kathleen A Hill,Paris Margaritis,Katherine A High,Steve S Sommer,

Aminoglycosides can bypass nonsense mutations and are the prototypic agents for translational bypass therapy (TBT). Initial results demonstrate the need for more potent drugs and an in vivo model system for quantitative assessment of TBT. Herein, we present an in vivo system for evaluating the efficacy of premature stop codon management therapies: in vivo quantitative stop codon management repli-sampling TBT efficacy assay (IQSCMaRTEA). Application of IQSCMaRTEA reveals that geneticin is much more efficacious in vivo than gentamicin. Treatment with geneticin elicits a multiday response, and residual F9 antigen can be detected after 3 weeks. These data demonstrate the utility of IQSCMaRTEA for evaluating drugs that bypass nonsense mutations. In addition, IQSCMaRTEA may be helpful for testing inhibitors of nonsense-mediated decay, as stop codon management therapy will sometimes require inhibition of nonsense-mediated decay and translational bypass of the nonsense mutation. Furthermore, geneticin, its metabolites, or better tolerated analogues should be evaluated as a general treatment with multiday response for severe genetic disease caused by nonsense mutation.

A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin. Publishing Authors By Initials

C Yang,J Feng,W Song,J Wang,B Tsai,Y Zhang,WA Scaringe,KA Hill,P Margaritis,KA High,SS Sommer,

For similar genetic processes: gene expression: protein biosynthesis research abstracts see: genetic processes: gene expression: protein biosynthesis research

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A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin. Journal Published:

PUBLICATION TYPE: Research Support, Non-U.S. Gov

Journal: Proceedings of the National Academy of Sciences of

VOLUME: 104

Page Numbers: 15394-9

Journal Abbreviation: Proc. Natl. Acad. Sci. U.S.A.

ISSN: 0027-8424

DAY: 19

MONTH: 09

YEAR: 2007

A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin. Information

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LANGUAGE: eng

NlmUniqueID: 7505876

A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin. Keywords Mesh Terms:

KEYWORDS: Protein Biosynthesis

MESH TERMS: metabolism

Chemical & Substance for Abstract: A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin. Information

Substance Name: antibiotic G 418

Registry Number: 49863-47-0

Grant and Affiliation Information for A mouse model for nonsense mutation bypass therapy shows a dramatic multiday response to geneticin.

AFFILIATION: Department of Molecular Genetics, City of Hope National Medical Center, Duarte, CA 91010, USA.

Country: United States

AGENCY: United States NHLBI

GRANT: P01HL074124

ACRONYM: HL

MEDLINETA: Proc Natl Acad Sci U S A

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