Increased systemic HSP70B levels in spinal muscular atrophy infants

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...

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Autores principales: Eichelberger, Eric J., Alves, Christiano R. R., Zhang, Ren, Petrillo, Marco, Cullen, Patrick, Farwell, Wildon, Hurt, Jessica A., Staropoli, John F., Swoboda, Kathryn J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Brief Communications
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8283166/
https://www.ncbi.nlm.nih.gov/pubmed/33991176
http://dx.doi.org/10.1002/acn3.51377
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author Eichelberger, Eric J.
Alves, Christiano R. R.
Zhang, Ren
Petrillo, Marco
Cullen, Patrick
Farwell, Wildon
Hurt, Jessica A.
Staropoli, John F.
Swoboda, Kathryn J.
author_facet Eichelberger, Eric J.
Alves, Christiano R. R.
Zhang, Ren
Petrillo, Marco
Cullen, Patrick
Farwell, Wildon
Hurt, Jessica A.
Staropoli, John F.
Swoboda, Kathryn J.
author_sort Eichelberger, Eric J.
collection PubMed
description 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.
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spelling pubmed-82831662021-07-21 Increased systemic HSP70B levels in spinal muscular atrophy infants Eichelberger, Eric J. Alves, Christiano R. R. Zhang, Ren Petrillo, Marco Cullen, Patrick Farwell, Wildon Hurt, Jessica A. Staropoli, John F. Swoboda, Kathryn J. Ann Clin Transl Neurol Brief Communications 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. John Wiley and Sons Inc. 2021-05-15 /pmc/articles/PMC8283166/ /pubmed/33991176 http://dx.doi.org/10.1002/acn3.51377 Text en © 2021 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
spellingShingle Brief Communications
Eichelberger, Eric J.
Alves, Christiano R. R.
Zhang, Ren
Petrillo, Marco
Cullen, Patrick
Farwell, Wildon
Hurt, Jessica A.
Staropoli, John F.
Swoboda, Kathryn J.
Increased systemic HSP70B levels in spinal muscular atrophy infants
title Increased systemic HSP70B levels in spinal muscular atrophy infants
title_full Increased systemic HSP70B levels in spinal muscular atrophy infants
title_fullStr Increased systemic HSP70B levels in spinal muscular atrophy infants
title_full_unstemmed Increased systemic HSP70B levels in spinal muscular atrophy infants
title_short Increased systemic HSP70B levels in spinal muscular atrophy infants
title_sort increased systemic hsp70b levels in spinal muscular atrophy infants
topic Brief Communications
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8283166/
https://www.ncbi.nlm.nih.gov/pubmed/33991176
http://dx.doi.org/10.1002/acn3.51377
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