iPSC for modeling neurodegenerative disorders

Regen Ther. 2020 Dec 11:15:332-339. doi: 10.1016/j.reth.2020.11.006. eCollection 2020 Dec.

Abstract

Neurodegenerative disorders such as Parkinson's and Alzheimer's disease, are fundamental health concerns all around the world. The development of novel treatments and new techniques to address these disorders, are being actively studied by researchers and medical personnel. In the present review we will discuss the application of induced Pluripotent Stem Cells (iPSCs) for cell-therapy replacement and disease modelling. The aim of iPSCs is to restore the functionality of the damaged tissue by replacing the impaired cells with competitive ones. To achieve this objective, iPSCs can be properly differentiated into virtually any cell fate and can be strongly translated into human health via in vitro and in vivo disease modeling for the development of new therapies, the discovery of biomarkers for several disorders, the elaboration and testing of new drugs as novel treatments, and as a tool for personalized medicine.

Keywords: AD, Alzheimer's disease; AFP, Alpha-Fetoprotein; Alzheimer; Aβ, β-Amyloid; B-III-TUB, β–III–Tubulin; BBB, Blood Brain Barrier; CRISPR, Clustered Regularly Interspaced Short Palindromic Repeats; DOPAL, 3,4-Dihydroxyphenylacetaldehyde; EBs, Embryoid Bodies; FLASH, Fast Length Adjustment of Short Reads; LUHMES, Lund Human Mesencephalic Cell Line; MHC, Mayor Histocompatibility Complex; Neurodegenerative diseasaes; PCR, Polymerase Chain Reaction; PD, Parkinson's Disease; Parkinson; ROS, Reactive Oxygen Species; SCs, Stem Cells; SMA, Smooth-Muscle Antibody; SNPc, Substantia Nigra Pars Compacta; TH, Tyrosine Hydroxylase; WGS, Whole Genome Sequencing; gRNA, guide RNA; hESC, Human Embryonic Stem Cells; iPSCs; iPSCs, Induced Pluripotent Stem Cells; nsSNVs, nonsynonymous single nucleotide variants; pTau, Phosphorylated Tau.

Publication types

  • Review