Increased systemic HSP70B levels in spinal muscular atrophy infants.

Eric J Eichelberger, Christiano R R Alves, Ren Zhang, Marco Petrillo, Patrick Cullen, Wildon Farwell, Jessica A Hurt, John F Staropoli, Kathryn J Swoboda

Annals of clinical and translational neurology 2021 Jul

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Despite newly available treatments for spinal muscular atrophy (SMA), novel circulating biomarkers are still critically necessary to track SMA progression and therapeutic response. To identify potential biomarkers, we performed whole-blood RNA sequencing analysis in SMA type 1 subjects under 1 year old and age-matched healthy controls. Our analysis revealed the Heat Shock Protein Family A Member 7 (HSPA7)/heat shock 70kDa protein 7 (HSP70B) as a novel candidate biomarker to track SMA progression early in life. Changes in circulating HSP70B protein levels were associated with changes in circulating neurofilament levels in SMA newborns and infants. Future studies will determine whether HSP70B levels respond to molecular therapies. © 2021 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.

Citation

Eric J Eichelberger, Christiano R R Alves, Ren Zhang, Marco Petrillo, Patrick Cullen, Wildon Farwell, Jessica A Hurt, John F Staropoli, Kathryn J Swoboda. Increased systemic HSP70B levels in spinal muscular atrophy infants. Annals of clinical and translational neurology. 2021 Jul;8(7):1495-1501