Editorial: Gene Therapy in the CNS - Progress and Prospects for Novel Therapies.
作者信息
Ledri Marco, Sørensen Andreas T, Kokaia Merab, Woldbye David P D, Gøtzsche Casper R
机构信息
Laboratory of Molecular Neurophysiology and Epilepsy, Department of Clinical Sciences, Epilepsy Center, Faculty of Medicine, Lund University, Lund, Sweden.
Department of Neuroscience, University of Copenhagen, Copenhagen, Denmark.
出版信息
Front Mol Neurosci. 2021 Oct 20;14:778134. doi: 10.3389/fnmol.2021.778134. eCollection 2021.
Abstract
摘要
相似文献
1
Editorial: Gene Therapy in the CNS - Progress and Prospects for Novel Therapies.
Front Mol Neurosci. 2021 Oct 20;14:778134. doi: 10.3389/fnmol.2021.778134. eCollection 2021.
2
Gene Therapy with CRISPR/Cas9 Coming to Age for HIV Cure.基因治疗与 CRISPR/Cas9 渐趋成熟，有望攻克 HIV。
AIDS Rev. 2017 Oct-Dec;19(3):167-172.
3
Combining Engineered Nucleases with Adeno-associated Viral Vectors for Therapeutic Gene Editing.将工程核酸酶与腺相关病毒载体相结合用于治疗性基因编辑。
Adv Exp Med Biol. 2017;1016:29-42. doi: 10.1007/978-3-319-63904-8_2.
4
A Self-Deleting AAV-CRISPR System for Genome Editing.一种用于基因组编辑的自我删除型腺相关病毒-成簇规律间隔短回文重复序列系统
Mol Ther Methods Clin Dev. 2018 Dec 6;12:111-122. doi: 10.1016/j.omtm.2018.11.009. eCollection 2019 Mar 15.
5
Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice.腺相关病毒介导的CRISPR在小鼠心脏基因编辑中的递送
J Vis Exp. 2018 Aug 2(138):57560. doi: 10.3791/57560.
6
genome editing in animals using AAV-CRISPR system: applications to translational research of human disease.使用AAV-CRISPR系统对动物进行基因组编辑：在人类疾病转化研究中的应用
F1000Res. 2017 Dec 20;6:2153. doi: 10.12688/f1000research.11243.1. eCollection 2017.
7
Genome Editing for CNS Disorders.用于中枢神经系统疾病的基因组编辑
Front Neurosci. 2020 Oct 22;14:579062. doi: 10.3389/fnins.2020.579062. eCollection 2020.
8
Sendai virus, an RNA virus with no risk of genomic integration, delivers CRISPR/Cas9 for efficient gene editing.仙台病毒，一种没有基因组整合风险的 RNA 病毒，可递送 CRISPR/Cas9 进行高效的基因编辑。
Mol Ther Methods Clin Dev. 2016 Aug 24;3:16057. doi: 10.1038/mtm.2016.57. eCollection 2016.
9
Progress and prospects in plant genome editing.植物基因组编辑的进展与展望。
Nat Plants. 2017 Jul 31;3:17107. doi: 10.1038/nplants.2017.107.
10
Delivery Platforms for CRISPR/Cas9 Genome Editing of Glial Cells in the Central Nervous System.用于中枢神经系统胶质细胞CRISPR/Cas9基因组编辑的递送平台
Front Genome Ed. 2021 Mar 3;3:644319. doi: 10.3389/fgeed.2021.644319. eCollection 2021.
本文引用的文献
1
Gene specific therapies - the next therapeutic milestone in neurology.基因特异性疗法——神经病学的下一个治疗里程碑。
Neurol Res Pract. 2020 Sep 8;2:25. doi: 10.1186/s42466-020-00075-z. eCollection 2020.
2
Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence.视网膜疾病中的voretigene neparvovec：当前临床证据综述
Clin Ophthalmol. 2020 Nov 13;14:3855-3869. doi: 10.2147/OPTH.S231804. eCollection 2020.
3
The once and future gene therapy.曾经与未来的基因治疗。
Nat Commun. 2020 Nov 16;11(1):5820. doi: 10.1038/s41467-020-19505-2.
4
Regulators' Advice Can Make a Difference: European Medicines Agency Approval of Zynteglo for Beta Thalassemia.监管机构的建议能产生影响：欧洲药品管理局批准Zynteglo用于治疗β地中海贫血。
Clin Pharmacol Ther. 2020 Mar;107(3):492-494. doi: 10.1002/cpt.1639. Epub 2019 Nov 8.
5
Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?基于 Luxturna（以及 Zolgensma 和 Glybera）的腺相关病毒基因治疗的现状：我们在哪里，我们是如何走到这一步的？
Annu Rev Virol. 2019 Sep 29;6(1):601-621. doi: 10.1146/annurev-virology-092818-015530. Epub 2019 Jul 5.
6
Gene therapy for ADA-SCID, the first marketing approval of an gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products.用于 ADA-SCID 的基因治疗，欧洲首个获得营销批准的基因治疗药物：为新一代先进治疗药物铺平道路。
EMBO Mol Med. 2017 Jun;9(6):737-740. doi: 10.15252/emmm.201707573.
7
Re-examination of regulatory opinions in Europe: possible contribution for the approval of the first gene therapy product Glybera.重新审视欧洲的监管意见：可能为首个基因治疗产品 Glybera 的批准做出贡献。
Mol Ther Methods Clin Dev. 2015 Feb 11;2:14066. doi: 10.1038/mtm.2014.66. eCollection 2015.
Zotero 插件