Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease-Reference-Cited by-同舟云学术

Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease

Published:2021-09-01 Issue:17-18 Volume:32 Page:949-958
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Schejtman Andrea¹,Vetharoy Winston¹,Choi Uimook²,Rivat Christine³,Theobald Narda²,Piras Giuseppa¹,Leon-Rico Diego¹,Buckland Karen¹,Armenteros-Monterroso Elena¹,Benedetti Sara¹,Ashworth Michael T.⁴,Rothe Michael⁵,Schambach Axel⁵⁶,Gaspar Hubert Bobby⁷,Kang Elizabeth M.²,Malech Harry L.²,Thrasher Adrian J.¹³,Santilli Giorgia¹

Affiliation:

1. Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.

2. Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.

3. Great Ormond Street Hospital for Children, NHS Foundation Trust, London, United Kingdom.

4. Department of Histopathology, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, United Kingdom.

5. Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany.

6. Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.

7. Orchard Therapeutics, London, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2020.276

Reference29 articles.

1. A Review of Chronic Granulomatous Disease

2. Chronic Granulomatous Disease: Epidemiology, Pathophysiology, and Genetic Basis of Disease

3. Clinical, functional, and genetic characterization of chronic granulomatous disease in 89 Turkish patients

4. Genetic and molecular findings of 38 Iranian patients with chronic granulomatous disease caused by p47‐phoxdefect

5. Chronic granulamatous disease: Two decades of experience from a paediatric immunology unit in a country with high rate of consangineous marriages

Cited by 5 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. Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy;Molecular Therapy - Methods & Clinical Development;2024-06

2. Gene Therapy for Inborn Errors of Immunity;The Journal of Allergy and Clinical Immunology: In Practice;2023-06

3. Chronic Granulomatous Disease;NADPH Oxidases Revisited: From Function to Structure;2023

4. Definitive Treatments for Chronic Granulomatous Disease with a Focus on Gene Therapy;NADPH Oxidases Revisited: From Function to Structure;2023

5. Gene therapy for inborn error of immunity – current status and future perspectives;Current Opinion in Allergy & Clinical Immunology;2022-12-06