基因治疗在罕见遗传性神经发育性精神障碍中的应用。
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.
机构信息
Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Horae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, UMass Chan Medical School, Worcester, MA, USA.
出版信息
Mol Ther. 2022 Jul 6;30(7):2416-2428. doi: 10.1016/j.ymthe.2022.05.014. Epub 2022 May 17.
We are in an emerging era of gene-based therapeutics with significant promise for rare genetic disorders. The potential is particularly significant for genetic central nervous system disorders that have begun to achieve Food and Drug Administration approval for select patient populations. This review summarizes the discussions and presentations of the National Institute of Mental Health-sponsored workshop "Gene-Based Therapeutics for Rare Genetic Neurodevelopmental Psychiatric Disorders," which was held in January 2021. Here, we distill the points raised regarding various precision medicine approaches related to neurodevelopmental and psychiatric disorders that may be amenable to gene-based therapies.
我们正处在一个基于基因的治疗新时代，这为罕见遗传病带来了巨大的希望。对于已经开始获得美国食品和药物管理局批准用于特定患者群体的遗传性中枢神经系统疾病，这种潜力尤为显著。本综述总结了由美国国立精神卫生研究所主办的研讨会“用于罕见遗传性神经发育性精神疾病的基因治疗”的讨论和报告，该研讨会于 2021 年 1 月举行。在这里，我们提炼了与可能适合基因治疗的神经发育和精神疾病相关的各种精准医学方法所提出的要点。
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