13C/31P MRS Metabolic Biomarkers of Disease Progression and Response to AAV Delivery of hGAA in a Mouse Model of Pompe Disease-Reference-Cited by-同舟云学术

登录注册会员服务联系我们

13C/31P MRS Metabolic Biomarkers of Disease Progression and Response to AAV Delivery of hGAA in a Mouse Model of Pompe Disease

Published:2017-12 Issue: Volume:7 Page:42-49
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Baligand Celine,Todd Adrian G.,Lee-McMullen Brittany,Vohra Ravneet S.,Byrne Barry J.,Falk Darin J.,Walter Glenn A.

Funder

Muscular Dystrophy Association

National High Magnetic Field Laboratory

Southeast Center for Integrative Metabolomics

NIAMS

National Science Foundation

State of Florida

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference37 articles.

1. Glycogen storage disease type II: acid alpha-glucosidase (acid maltase) deficiency;Hirschhorn,2001

2. Pompe disease: design, methodology, and early findings from the Pompe Registry;Byrne;Mol. Genet. Metab.,2011

3. Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes;Smith;Hum. Gene Ther.,2013

4. The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?;Lachmann;Orphanet J. Rare Dis.,2013

5. 13C NMR of intermediary metabolism: implications for systemic physiology;Shulman;Annu. Rev. Physiol.,2001

Cited by 10 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Non-invasive optoacoustic imaging of glycogen-storage and muscle degeneration in late-onset Pompe disease;Nature Communications;2024-09-08

2. From Acid Alpha-Glucosidase Deficiency to Autophagy: Understanding the Bases of POMPE Disease;International Journal of Molecular Sciences;2023-08-05

3. Lysosomal glycogen accumulation in Pompe disease results in disturbed cytoplasmic glycogen metabolism;Journal of Inherited Metabolic Disease;2022-10-17

4. High‐resolution magic angle spinning NMR for intact biological specimen analysis: Initial discovery, recent developments, and future directions;NMR in Biomedicine;2022-02-08

5. Further perspectives and challenges;Diabetes Without Needles;2022

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线，采集、加工和组织学术论文而形成的新型学术文献查询和分析系统，可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容，当前同舟云学术共收录了国内外主流学术期刊6万余种，收集的期刊论文及会议论文总量共计约1.5亿篇，并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询！咨询电话：010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号京ICP备18003416号-3