Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec-Reference-Cited by-同舟云学术

Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec

Published:2023-10 Issue:10 Volume:31 Page:2991-2998
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Retson Laura,Tiwari Nishant,Vaughn Jennifer,Bernes Saunder,Adelson P. David,Mansfield Keith,Libertini Silvana,Kuzmiski Brent,Alecu Iulian,Gabriel Richard,Mangum Ross^ORCID

Funder

Novartis Gene Therapies

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference39 articles.

1. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy;Lorson;Proc. Natl. Acad. Sci. USA,1999

2. Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2;Mailman;Genet. Med.,2002

3. Correlation between SMA type and SMN2 copy number revisited: An analysis of 625 unrelated Spanish patients and a compilation of 2834 reported cases;Calucho;Neuromuscul. Disord.,2018

4. Observational study of spinal muscular atrophy type I and implications for clinical trials;Finkel;Neurology,2014

5. Natural history of infantile-onset spinal muscular atrophy;Kolb;Ann. Neurol.,2017

Cited by 11 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. Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment;Orphanet Journal of Rare Diseases;2024-09-13

2. The evolving landscape of gene therapy for congenital severe hemophilia: a 2024 state of the art;Bleeding, Thrombosis and Vascular Biology;2024-09-04

3. Meeting Report: 2023 Muscular Dystrophy Association Summit on ‘Safety and Challenges in Gene Therapy of Neuromuscular Diseases’;Journal of Neuromuscular Diseases;2024-09-03

4. Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article;Human Gene Therapy;2024-08-01

5. Clinical perspectives: Treating spinal muscular atrophy;Molecular Therapy;2024-08