A systematic review of adeno-associated virus gene therapies in neurology: the need for consistent safety monitoring of a promising treatment
A systematic review of adeno-associated virus gene therapies in neurology: the need for consistent safety monitoring of a promising treatment
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神经病学腺相关病毒基因疗法的系统评价：需要对有前途的治疗进行持续的安全监测
DOI：
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发表时间：
2022
影响因子：
11
通讯作者：
L. Servais
中科院分区：
文献类型：
--
作者：
Rebecca H Horton;D. Saade;Theodora Markati;E. Harriss;C. Bönnemann;F. Muntoni;L. Servais
腺相关病毒（AAV）基因疗法在罕见疾病领域引起了极大的兴奋，特别是对于以前无法治疗的神经系统疾病。一些基因治疗产品已被宣称有效，试验的数量正在迅速增加。然而，正在出现与治疗有关的严重不良反应的报告，包括死亡。对其病因、预防和治疗的认识仍然不足。因此，我们承诺系统地审查有关AAV基因治疗的公开可用数据，以便整理有关安全性和有效性的现有信息。在这里，我们回顾了这些新疗法的疗效报告，其中许多显示出希望。我们还整理了越来越多的不良反应。压倒性地，这些结果为统一报告不良事件提供了理由。这可能对提高这些有前景的治疗方法的安全性至关重要。
Adeno-associated virus (AAV) gene therapies are generating much excitement in the rare disease field, particularly for previously untreatable neurological conditions. Efficacy has been claimed for several gene therapy products and the number of trials is rapidly increasing. However, reports of severe treatment-related adverse reactions are emerging, including death. There is still insufficient knowledge about their aetiology, prevention and treatment. We therefore undertook to systematically review publicly available data on AAV gene therapies in order to collate existing information on both safety and efficacy. Here, we review emerging efficacy reports of these novel therapies, many of which show promise. We also collate an increasing number of adverse reactions. Overwhelmingly, these results make a case for unified reporting of adverse events. This is likely to be critical for improving the safety of these promising treatments.
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影响因子：
4.2
作者：
Worgall, Stefan;Sondhi, Dolan;Crystal, Ronald G.
通讯作者：
Crystal, Ronald G.
影响因子：
4.2
作者：
Hinderer, Christian;Katz, Nathan;Wilson, James M.
通讯作者：
Wilson, James M.
影响因子：
12.4
作者：
George, Lindsey A.;Ragni, Margaret, V;High, Katherine A.
通讯作者：
High, Katherine A.
DOI：
10.1056/nejmoa1407309
发表时间：
2014-11-20
期刊：
The New England journal of medicine
影响因子：
--
作者：
Nathwani AC;Reiss UM;Tuddenham EG;Rosales C;Chowdary P;McIntosh J;Della Peruta M;Lheriteau E;Patel N;Raj D;Riddell A;Pie J;Rangarajan S;Bevan D;Recht M;Shen YM;Halka KG;Basner-Tschakarjan E;Mingozzi F;High KA;Allay J;Kay MA;Ng CY;Zhou J;Cancio M;Morton CL;Gray JT;Srivastava D;Nienhuis AW;Davidoff AM
通讯作者：
Davidoff AM
DOI：
10.1056/nejmoa1108046
发表时间：
2011-12-22
期刊：
The New England journal of medicine
影响因子：
--
作者：
Nathwani AC;Tuddenham EG;Rangarajan S;Rosales C;McIntosh J;Linch DC;Chowdary P;Riddell A;Pie AJ;Harrington C;O'Beirne J;Smith K;Pasi J;Glader B;Rustagi P;Ng CY;Kay MA;Zhou J;Spence Y;Morton CL;Allay J;Coleman J;Sleep S;Cunningham JM;Srivastava D;Basner-Tschakarjan E;Mingozzi F;High KA;Gray JT;Reiss UM;Nienhuis AW;Davidoff AM
通讯作者：
Davidoff AM