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RNA Quality Control and Protein Aggregates in Amyotrophic Lateral Sclerosis: A Review

  • Journal Review by Ashok Verma, MD, DM, MBA, and Rup Tandan, MD, FRCP

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  • Language: English
  • Published: 5/17/2013
  • Description: Available to Members ONLY - Members Free CME Amyotrophic lateral sclerosis (ALS) is the most common motor neuron disease in adults. The biologic basis of ALS remains unknown. However, ALS research has taken a dramatic turn over the past 4 years. Ground breaking discoveries of gene mutations that encode RNA processing proteins, and demonstration that abnormal aggregates of these and other proteins precede motor neuron loss in familial and sporadic ALS, have initiated a paradigm shift in understanding the pathogenic mechanisms of ALS. Curiously, some of these RNA binding proteins have prion-like domains, with a propensity to self-aggregation. The emerging hypothesis that a focal cascade of toxic protein aggregates, and their consequent non–cell-autonomous spread to neighborhood groups of neurons, fits the classical temporo-spatial progression of ALS. This article reviews the current research efforts toward understanding the role of RNA-processing regulation and protein aggregates in ALS.

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    ACCREDITATION STATEMENT
    The AANEM is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
    CREDIT DESIGNATION
    The AANEM designates this enduring material for a maximum of 2 AMA PRA Category 1 Credits TM. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Credit expires 5/31/2019.
    DISCLOSURE INFORMATION
    No one involved in the planning of this CME activity had any relevant financial relationships to disclose. Any conflicts of interest have been resolved by the Journal in accordance with the Muscle & Nerve editorial process.
    FORMAT: PDF

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