Salmeterol with Liver Depot Gene Therapy Enhances the Skeletal Muscle Response in Murine Pompe Disease-Reference-Cited by-同舟云学术

Salmeterol with Liver Depot Gene Therapy Enhances the Skeletal Muscle Response in Murine Pompe Disease

Published:2019-07 Issue:7 Volume:30 Page:855-864
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Han Sang-oh¹,Li Songtao¹,Everitt Jeffrey I.²,Koeberl Dwight D.¹³

Affiliation:

1. Division of Medical Genetics, Department of Pediatrics, Duke University Medical School, Durham, North Carolina.

2. Department of Pathology, Duke University Medical School, Durham, North Carolina.

3. Department of Molecular Genetics and Metabolism, Duke University Medical School, Durham, North Carolina.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2018.197

Reference38 articles.

1. 1. HirschhornR, ReuserAJJ. Glyogen storage disease type II: Acid α-glucosidase (acid maltase) deficiency: ScriverCR, BeaudetAL, SlyWS, ValleD, eds. The Metabolic and Molecular Basis for Inherited Disease, 8th ed. New York: Mcgraw-Hill, 2001:3389–3419.

2. Recombinant human acid -glucosidase: Major clinical benefits in infantile-onset Pompe disease

3. Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease

4. Oropharyngeal Dysphagia in Infants and Children with Infantile Pompe Disease

5. Clinical and Histologic Ocular Findings in Pompe Disease

Cited by 12 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. Sympathetic Nerves Coordinate Corneal Epithelial Wound Healing by Controlling the Mobilization of Ly6C^hi Monocytes From the Spleen to the Injured Cornea;Investigative Opthalmology & Visual Science;2023-09-08

2. Phase I study of liver depot gene therapy in late-onset Pompe disease;Molecular Therapy;2023-07

3. Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies;International Journal of Molecular Sciences;2022-06-04

4. Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease;Human Gene Therapy;2022-05-01

5. What’s new and what’s next for gene therapy in Pompe disease?;Expert Opinion on Biological Therapy;2022-04-27