Towards Advanced iPSC-based Drug Development for Neurodegenerative Disease

被引：34

作者：

Pasteuning-Vuhman, Svetlana ^{[1
]}

de Jongh, Rianne ^{[1
]}

Timmers, Annabel ^{[1
]}

Pasterkamp, R. Jeroen ^{[1
]}

机构：

[1] Univ Utrecht, Univ Med Ctr, Dept Translat Neurosci, Utrecht Brain Ctr, Utrecht, Netherlands

来源：

TRENDS IN MOLECULAR MEDICINE | 2021年 / 27卷 / 03期

关键词：

FAMILIAL ALZHEIMERS-DISEASE; BLOOD-BRAIN-BARRIER; 3-DIMENSIONAL CELL-CULTURE; STEAROYL-COA DESATURASE; MOTOR-NEURONS; STEM-CELLS; MODEL; ALS; REVEALS; INHIBITION;

D O I：

10.1016/j.molmed.2020.09.013

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Neurodegenerative diseases (NDDs) are a heterogeneous group of diseases that are characterized by the progressive loss of neurons leading to motor, sensory, and/or cognitive defects. Currently, NDDs are not curable and treatment focuses on alleviating symptoms and halting disease progression. Phenotypic heterogeneity between individual NDD patients, lack of robust biomarkers, the limited translational potential of experimental models, and other factors have hampered drug development for the treatment of NDDs. This review summarizes and discusses the use of induced pluripotent stem cell (iPSC) approaches for improving drug discovery and testing. It highlights challenges associated with iPSC model ing and also discusses innovative approaches such as brain organoids and microfluidic-based technology which will improve drug development for NDDs.

引用

页码：263 / 279

页数：17

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