What is the clinical evidence from this study?

Study design: Other. Population: Myotonic dystrophy I (DM1). Intervention: Expanded CUG repeats vs. Normal myoblasts. Primary outcome: Stoichiometry of MBNL1(CUG) complexes and RNA splicing.

What does this research mean for the field?

Expression of expanded CUG repeats dysregulates RNA splicing in myotonic dystrophy type 1 by altering the stoichiometry of MBNL1 complexes through increased levels of partner proteins. Novelty: ClaimNovelty.NOVEL_FINDING. Consensus alignment: ConsensusAlignment.NEUTRAL.

September 8, 2011Journal of Biological ChemistryOpen Access

Expanded CUG Repeats Dysregulate RNA Splicing by Altering the Stoichiometry of the Muscleblind 1 Complex

View Full Paper

Ask AI

Bookmark

Key result

Expanded CUG repeats alter MBNL1 complex stoichiometry in DM1 myoblasts, dysregulating RNA splicing.

Population

Normal human myoblasts, myotonic dystrophy I (DM1) myoblasts, and Cos7 cells

Comparison

Expression of expanded CUG repeat RNA and… vs Normal myoblasts or baseline state

Design

Preclinical

Authors

SPSharan PaulUniversity of Utah WDWarunee DansithongUniversity of Utah SJSonali P. JogUniversity of Southern California

Discussion

Member takes

Implication

Should not yet inform DM1 management; leaves open MBNL1 stoichiometry as a therapeutic target in models.

Structured PICO

Population

Normal human myoblasts, myotonic dystrophy I (DM1) myoblasts, and Cos7 cells

Exposure

Expression of expanded CUG repeat RNA and modeling increased levels of hnRNP H, H2, H3, F, and DDX5

Comparator

Normal myoblasts or baseline state

Outcome

Stoichiometry of MBNL1(CUG) complexes and RNA splicing dysregulationsurrogate

Expanded CUG repeats in myotonic dystrophy type 1 alter the stoichiometry of MBNL1 complexes, leading to the reinforcement and expansion of RNA processing defects.

Cite This Study

Paul et al. (2011) studied Myotonic dystrophy I (DM1). Expanded CUG repeats vs. Normal myoblasts was evaluated on Stoichiometry of MBNL1(CUG) complexes and RNA splicing. Expression of expanded CUG repeats altered the stoichiometry of MBNL1(CUG) complexes in DM1 myoblasts, increasing levels of partner proteins and dysregulating RNA splicing.

synapsesocial.com/papers/6aa7a3eeea76de5c364f28a2 https://doi.org/10.1074/jbc.m111.255224

View Full Paper

Ask AI

Bookmark

September 8, 2011Journal of Biological ChemistryOpen Access

Expanded CUG Repeats Dysregulate RNA Splicing by Altering the Stoichiometry of the Muscleblind 1 Complex

View Full Paper

Ask AI

Bookmark

Key result

Expanded CUG repeats alter MBNL1 complex stoichiometry in DM1 myoblasts, dysregulating RNA splicing.

Population

Normal human myoblasts, myotonic dystrophy I (DM1) myoblasts, and Cos7 cells

Comparison

Expression of expanded CUG repeat RNA and… vs Normal myoblasts or baseline state

Design

Preclinical

Authors

SPSharan PaulUniversity of Utah WDWarunee DansithongUniversity of Utah SJSonali P. JogUniversity of Southern California

Discussion

Member takes

Implication

Should not yet inform DM1 management; leaves open MBNL1 stoichiometry as a therapeutic target in models.

Structured PICO

Population

Normal human myoblasts, myotonic dystrophy I (DM1) myoblasts, and Cos7 cells

Exposure

Expression of expanded CUG repeat RNA and modeling increased levels of hnRNP H, H2, H3, F, and DDX5

Comparator

Normal myoblasts or baseline state

Outcome

Stoichiometry of MBNL1(CUG) complexes and RNA splicing dysregulationsurrogate

Expanded CUG repeats in myotonic dystrophy type 1 alter the stoichiometry of MBNL1 complexes, leading to the reinforcement and expansion of RNA processing defects.

Cite This Study

synapsesocial.com/papers/6aa7a3eeea76de5c364f28a2 https://doi.org/10.1074/jbc.m111.255224

Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

View Full Paper

Ask AI

Bookmark