Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead-Reference-Cited by-同舟云学术

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Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead

Published:2021-01 Issue:1 Volume:31 Page:69-78
ISSN:0960-8966
Container-title:Neuromuscular Disorders
language:en
Short-container-title:Neuromuscular Disorders

Author:

Heslop Emma,Turner Cathy,Irvin Anna,Muntoni Francesco,Straub Volker,Guglieri Michela

Publisher

Elsevier BV

Subject

Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health

Reference13 articles.

1. Adeno associated virus vector as a platform for gene therapy delivery;Wang;Nat Rev Drug Discov,2019

2. Acute lymphoblastic leukemia. Clinical use of lentiviral vectors;Milone;Leukemia,2018

3. Codon optimization of the microdystrophin gene for Duchene muscular dystrophy gene therapy;Athanasopoulos;Methods Mol Biol,2011

4. Systemic AAV microdystrophin gene therapy for duchenne muscular dystrophy;Duan;Mol Ther,2018

5. Very mild muscular dystrophy associated with the deletion of 46% of dystrophin;England;Nature,1990

Cited by 7 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. Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy;Journal of Neuromuscular Diseases;2024-09-03

2. Poly(vinyl alcohol) (PVA) for gene delivery: a systematic review;Journal of Drug Delivery Science and Technology;2024-09

3. A Voyage on the Role of Nuclear Factor Kappa B (NF-kB) Signaling Pathway in Duchenne Muscular Dystrophy: An Inherited Muscle Disorder;Cureus;2024-08-27

4. A Novel CRISPR-Cas9 Strategy to Target DYSTROPHIN Mutations Downstream of Exon 44 in Patient-Specific DMD iPSCs;Cells;2024-06-04

5. Performing clinical drug trials in children with a rare disease;Essentials of Translational Pediatric Drug Development;2024

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