Drug Discovery of Spinal Muscular Atrophy (SMA) from the Computational Perspective: A Comprehensive Review.
Drug Discovery of Spinal Muscular Atrophy (SMA) from the Computational Perspective: A Comprehensive Review.
复制标题
从计算角度看脊髓性肌萎缩症(SMA)的药物发现：综述。
DOI：
10.3390/ijms22168962
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发表时间：
2021-08-20
影响因子：
5.6
通讯作者：
Choi SB
中科院分区：
文献类型：
--
作者：
Chong LC;Gandhi G;Lee JM;Yeo WWY;Choi SB
脊髓性肌萎缩症（SMA）是儿童死亡的主要遗传原因之一，是一种罕见的神经肌肉疾病，由编码SMN蛋白的存活运动神经元1 （SMN1）基因的功能丧失突变引起。当神经元中缺乏SMN蛋白时，患者会出现肌肉无力和萎缩，严重者会出现呼吸衰竭和死亡。几种治疗方法在人体试验中显示出希望，迄今为止，美国食品和药物管理局（FDA）已经批准了三种药物。尽管这些被批准的疗法显示出了希望，但仍有一些关键的限制，其中最重要的是成本。fda批准的药物价格高昂，被列入世界上最昂贵的治疗药物之列。价格仍然远远超出可承受范围，并可能成为患者的负担。生物医学数据的蓬勃发展和计算方法的进步为SMA治疗发展开辟了新的可能性。本文重点介绍了计算机辅助方法的现状，包括计算机药物再利用、网络驱动药物发现以及人工智能（AI）辅助药物发现，并讨论了未来的前景。
Spinal muscular atrophy (SMA), one of the leading inherited causes of child mortality, is a rare neuromuscular disease arising from loss-of-function mutations of the survival motor neuron 1 (SMN1) gene, which encodes the SMN protein. When lacking the SMN protein in neurons, patients suffer from muscle weakness and atrophy, and in the severe cases, respiratory failure and death. Several therapeutic approaches show promise with human testing and three medications have been approved by the U.S. Food and Drug Administration (FDA) to date. Despite the shown promise of these approved therapies, there are some crucial limitations, one of the most important being the cost. The FDA-approved drugs are high-priced and are shortlisted among the most expensive treatments in the world. The price is still far beyond affordable and may serve as a burden for patients. The blooming of the biomedical data and advancement of computational approaches have opened new possibilities for SMA therapeutic development. This article highlights the present status of computationally aided approaches, including in silico drug repurposing, network driven drug discovery as well as artificial intelligence (AI)-assisted drug discovery, and discusses the future prospects.
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影响因子：
14.9
作者：
Avram S;Bologa CG;Holmes J;Bocci G;Wilson TB;Nguyen DT;Curpan R;Halip L;Bora A;Yang JJ;Knockel J;Sirimulla S;Ursu O;Oprea TI
通讯作者：
Oprea TI
影响因子：
3
作者：
Bisgin H;Liu Z;Fang H;Kelly R;Xu X;Tong W
通讯作者：
Tong W
影响因子：
9.8
作者：
Campbell, L;Potter, A;Davies, K
通讯作者：
Davies, K
影响因子：
9.3
作者：
Bowerman M;Murray LM;Boyer JG;Anderson CL;Kothary R
通讯作者：
Kothary R
影响因子：
5
作者：
Butchbach ME
通讯作者：
Butchbach ME