Therapeutic targeting and HSC proliferation by small molecules and biologicals.

3区生物学 Q1 Biochemistry, Genetics and Molecular Biology Advances in protein chemistry and structural biology Pub Date : 2023-01-01 DOI:10.1016/bs.apcsb.2022.11.012

Esra Albayrak, Fatih Kocabaş

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Abstract

Hematopoietic stem cells (HSCs) have considerably therapeutic value on autologous and allogeneic transplantation for many malignant/non-malignant hematological diseases, especially with improvement of gene therapy. However, acquirement of limited cell dose from HSC sources is the main handicap for successful transplantation. Therefore, many strategies based on the utilization of various cytokines, interaction of stromal cells, modulation of several extrinsic and intrinsic factors have been developed to promote ex vivo functional HSC expansion with high reconstitution ability until today. Besides all these strategies, small molecules become prominent with their ease of use and various advantages when they are translated to the clinic. In the last two decades, several small molecule compounds have been investigated in pre-clinical studies and, some of them were evaluated in different stages of clinical trials for their safety and efficiencies. In this chapter, we will present an overview of HSC biology, function, regulation and also, pharmacological HSC modulation with small molecules from pre-clinical and clinical perspectives.

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小分子和生物制剂的治疗靶向和HSC增殖。

随着基因治疗技术的发展，造血干细胞在多种恶性/非恶性血液病的自体和异体移植中具有重要的治疗价值。然而，从造血干细胞来源获得有限的细胞剂量是成功移植的主要障碍。因此，迄今为止，许多基于各种细胞因子的利用、基质细胞的相互作用、几种外在和内在因素的调节的策略已经被开发出来，以促进具有高重构能力的离体功能性HSC扩增。除了这些策略外，小分子在转化为临床时，其易用性和各种优势变得突出。在过去的二十年中，一些小分子化合物已经在临床前研究中进行了研究，其中一些化合物在临床试验的不同阶段进行了安全性和有效性评估。在本章中，我们将从临床前和临床的角度概述HSC的生物学、功能、调控以及小分子对HSC的药理学调节。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Advances in protein chemistry and structural biology BIOCHEMISTRY & MOLECULAR BIOLOGY-

CiteScore

7.40

自引率

0.00%

发文量

审稿时长

>12 weeks

期刊介绍： Published continuously since 1944, The Advances in Protein Chemistry and Structural Biology series has been the essential resource for protein chemists. Each volume brings forth new information about protocols and analysis of proteins. Each thematically organized volume is guest edited by leading experts in a broad range of protein-related topics.