Emptying the stores: lysosomal diseases and therapeutic strategies

Emptying the stores: lysosomal diseases and therapeutic strategies, Published online: 17 November 2017; doi:10.1038/nrd.2017.214

NatureArticleSnippet(type=short-summary, markup=

Lysosomal storage disorders (LSDs) are rare, inherited metabolic disorders that result from defects in lysosomal function, for which treatment options are limited. Here, Platt provides an overview of LSDs, approved and potential therapeutic approaches and agents currently in development. The challenges associated with LSD diagnosis, drug development and treatment are discussed.

, isJats=true)

中文翻译：

---

清空商店：溶酶体疾病和治疗策略

清空商店：溶酶体疾病和治疗策略

清空商店：溶酶体疾病和治疗策略，在线发布：2017年11月17日；doi：10.1038 / nrd.2017.214

NatureArticleSnippet（type = short-summary，markup =

溶酶体贮积病（LSD）是由溶酶体功能缺陷导致的罕见的遗传性代谢紊乱，治疗选择受到限制。在这里，Platt概述了LSD，目前正在开发的批准的和潜在的治疗方法以及药物。讨论了与LSD诊断，药物开发和治疗相关的挑战。

，isJats = true）