Gene Replacement Therapy: A Primer for the Health-system Pharmacist.
Gene Replacement Therapy: A Primer for the Health-system Pharmacist.
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DOI：
10.1177/0897190019854962
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发表时间：
2020-12
影响因子：
1.3
通讯作者：
Blind J
中科院分区：
文献类型：
--
作者：
Petrich J;Marchese D;Jenkins C;Storey M;Blind J
基因替代治疗的综合评价，以及药师处理和管理的指导和专家意见。目前，全球有102600项基因治疗临床试验，美国有4种食品和药物管理局（FDA）批准的基因治疗产品。基因治疗及其处理与其他药物不同;然而，美国国立卫生研究院（NIH），FDA，疾病控制和预防中心（CDC），世界卫生组织（WHO）和专业协会缺乏关于其药物应用的指导。尽管NIH将基因治疗中病毒载体的主要生物制剂分为风险组，但关于最小化暴露和降低风险的信息不完整。在缺乏明确指导的情况下，各机构自行制定政策和程序，这些政策和程序往往各不相同，而且往往已经过时。这篇评论提供了专家意见的作用，药剂师在机构的准备，以及基因治疗的处理和管理。一个建议的基因替代治疗处理的基础设施模型，包括必要的设备购置和标准操作程序的发展。人员，病人和护理人员的教育和培训进行了讨论。药剂师在基因替代疗法的适当处理和一般管理、确定风险水平、建立基础设施以及制定适当的政策和方案方面发挥着关键作用，特别是在缺乏关于基因替代疗法处理和运输的共识指南的情况下。
Comprehensive review of gene replacement therapy with guidance and expert opinion on handling and administration for pharmacists. There are currently ∼2600 gene therapy clinical trials worldwide and 4 Food and Drug Administration (FDA)-approved gene therapy products available in the United States. Gene therapy and its handling are different from other drugs; however, there is a lack of guidance from the National Institutes of Health (NIH), FDA, Centers for Disease Control and Prevention (CDC), World Health Organization (WHO), and professional associations regarding their pharmaceutical application. Although the NIH stratifies the backbone biologicals of viral vectors in gene therapies into risk groups, incomplete information regarding minimization of exposure and reduction of risk exists. In the absence of defined guidance, individual institutions develop their own policies and procedures, which often differ and are often outdated. This review provides expert opinion on the role of pharmacists in institutional preparedness, as well as gene therapy handling and administration. A suggested infrastructural model for gene replacement therapy handling is described, including requisite equipment acquisition and standard operating procedure development. Personnel, patient, and caregiver education and training are discussed. Pharmacists have a key role in the proper handling and general management of gene replacement therapies, identifying risk level, establishing infrastructure, and developing adequate policies and protocols, particularly in the absence of consensus guidelines for the handling and transport of gene replacement therapies.
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影响因子：
46.9
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影响因子：
9.9
作者：
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通讯作者：
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作者：
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影响因子：
11.2
作者：
Kolb SJ;Coffey CS;Yankey JW;Krosschell K;Arnold WD;Rutkove SB;Swoboda KJ;Reyna SP;Sakonju A;Darras BT;Shell R;Kuntz N;Castro D;Parsons J;Connolly AM;Chiriboga CA;McDonald C;Burnette WB;Werner K;Thangarajh M;Shieh PB;Finanger E;Cudkowicz ME;McGovern MM;McNeil DE;Finkel R;Iannaccone ST;Kaye E;Kingsley A;Renusch SR;McGovern VL;Wang X;Zaworski PG;Prior TW;Burghes AHM;Bartlett A;Kissel JT;NeuroNEXT Clinical Trial Network on behalf of the NN101 SMA Biomarker Investigators
通讯作者：
NeuroNEXT Clinical Trial Network on behalf of the NN101 SMA Biomarker Investigators