National Survey on Clinical Practice of Gene Therapy using Adeno-associated Virus Vector ―The Actual Status and Hurdle on Clinical Practice in Gene Therapy for Spinal Muscular Atrophy―
Author:
Affiliation:
1. Gene and Cell Therapy Promotion Center, National Center for Child Health and Development, Japan
Publisher
Japanese Society of Clinical Pharmacology and Therapeutics
Subject
Pharmacology (medical),Pharmacology
Link
https://www.jstage.jst.go.jp/article/jscpt/53/6/53_217/_pdf
Reference18 articles.
1. 1) Ginn SL, Amaya AK, Alexander IE, Edelstein M, Abedi MR. Gene therapy clinical trials worldwide to 2017: an update. J Gene Med. 2018; 20(5): e3015. doi: 10.1002/jgm.3015. 2
2. 2) Blind JE, McLeod EN, Campbell KJ. Viral-mediated gene therapy and genetically modified therapeutics: a primer on biosafety handling for the health-system pharmacist. Am J Health Syst Pharm. 2019; 76(11): 795-802. doi: 10.1093/ajhp/zxz056.
3. 3) Petrich J, Marchese D, Jenkins C, Storey M, Blind J. Gene replacement therapy: a primer for the health-system pharmacist. J Pharm Pract. 2020; 33(6): 846-55. doi: 10.1177/0897190019854962.
4. 4) Maruyama Y, Sakurai A, Kasai M, Noda S, Honda F. Current status and future perspective of gene therapy products in Japan. Cell & Gene Therapy Insights. 2021; 7(3): 131-40, doi: 10.18609/cgti.2021.026.
5. 5) Ministry of the Environment. Biosafety Regulations in Japan.[https:// www.biodic.go.jp/bch/english/cartagena/index.html (accessed 2021-08-22)]
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