Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease-Reference-Cited by-同舟云学术

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Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease

Published:2022-05-01 Issue:9-10 Volume:33 Page:479-491
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Han Sang-oh¹,Gheorghiu Dorothy¹^ORCID,Chang Alex¹,Mapatano Sweet Hope¹,Li Songtao¹,Brooks Elizabeth¹²,Koeberl Dwight¹³

Affiliation:

1. Department of Pediatrics, Duke University School of Medicine, Durham, North Carolina, USA.

2. Department of Laboratory Animal Resources, Duke University, Durham, North Carolina, USA.

3. Department of Molecular Genetics and Microbiology, Duke University, Durham, North Carolina, USA.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2021.218

Reference32 articles.

1. Death from supine asphyxia in late onset pompe disease: Two patients

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

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

4. Clinical and Histologic Ocular Findings in Pompe Disease

Cited by 3 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. A Comprehensive Update on Late-Onset Pompe Disease;Biomolecules;2023-08-22

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

3. Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease;Gene Therapy;2023-01-11

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