P716: Post-translational processing of GAA protein associated with GAA variants may explain the clinical variability observed amongst Pompe disease patients

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Main Authors: Shelly Goomber, Catherine Rehder, Benjamin Cocanougher, Seung-Hye Jung, Gregory Crawford, Deeksha Bali, Priya Kishnani
Format: Article
Language:English
Published: Elsevier 2025-01-01
Series:Genetics in Medicine Open
Online Access:http://www.sciencedirect.com/science/article/pii/S2949774425011240
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author Shelly Goomber
Catherine Rehder
Benjamin Cocanougher
Seung-Hye Jung
Gregory Crawford
Deeksha Bali
Priya Kishnani
author_facet Shelly Goomber
Catherine Rehder
Benjamin Cocanougher
Seung-Hye Jung
Gregory Crawford
Deeksha Bali
Priya Kishnani
author_sort Shelly Goomber
collection DOAJ
format Article
id doaj-art-43c8e196ff284c4985a1a132ca124db7
institution DOAJ
issn 2949-7744
language English
publishDate 2025-01-01
publisher Elsevier
record_format Article
series Genetics in Medicine Open
spelling doaj-art-43c8e196ff284c4985a1a132ca124db72025-08-20T03:14:38ZengElsevierGenetics in Medicine Open2949-77442025-01-01310308510.1016/j.gimo.2025.103085P716: Post-translational processing of GAA protein associated with GAA variants may explain the clinical variability observed amongst Pompe disease patientsShelly Goomber0Catherine Rehder1Benjamin Cocanougher2Seung-Hye Jung3Gregory Crawford4Deeksha Bali5Priya Kishnani6Department of Pediatrics, Division of Medical Genetics, Duke Health, Durham, NCDepartment of Pathology, Duke Health, Durham, NCDepartment of Pediatrics, Division of Medical Genetics, Duke Health, Durham, NCDepartment of Pediatrics, Division of Medical Genetics, Duke Health, Durham, NCDepartment of Pediatrics, Division of Medical Genetics, Duke Health, Durham, NCDepartment of Pediatrics, Division of Medical Genetics, Duke Health, Durham, NCDepartment of Pediatrics, Division of Medical Genetics, Duke Health, Durham, NChttp://www.sciencedirect.com/science/article/pii/S2949774425011240
spellingShingle Shelly Goomber
Catherine Rehder
Benjamin Cocanougher
Seung-Hye Jung
Gregory Crawford
Deeksha Bali
Priya Kishnani
P716: Post-translational processing of GAA protein associated with GAA variants may explain the clinical variability observed amongst Pompe disease patients
Genetics in Medicine Open
title P716: Post-translational processing of GAA protein associated with GAA variants may explain the clinical variability observed amongst Pompe disease patients
title_full P716: Post-translational processing of GAA protein associated with GAA variants may explain the clinical variability observed amongst Pompe disease patients
title_fullStr P716: Post-translational processing of GAA protein associated with GAA variants may explain the clinical variability observed amongst Pompe disease patients
title_full_unstemmed P716: Post-translational processing of GAA protein associated with GAA variants may explain the clinical variability observed amongst Pompe disease patients
title_short P716: Post-translational processing of GAA protein associated with GAA variants may explain the clinical variability observed amongst Pompe disease patients
title_sort p716 post translational processing of gaa protein associated with gaa variants may explain the clinical variability observed amongst pompe disease patients
url http://www.sciencedirect.com/science/article/pii/S2949774425011240
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