Gene Therapy Using Neural Stem/Progenitor Cells Derived from Human Induced Pluripotent Stem Cells: Visualization of Migration and Bystander Killing Effect

2020 ◽  
Vol 31 (5-6) ◽  
pp. 352-366 ◽  
Author(s):  
Ryota Tamura ◽  
Hiroyuki Miyoshi ◽  
Yukina Morimoto ◽  
Yumiko Oishi ◽  
Oltea Sampetrean ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Induced Pluripotent Stem Cells ◽  
Progenitor Cells ◽  
Pluripotent Stem Cells ◽  
Killing Effect ◽  
Neural Stem Progenitor Cells ◽  
Bystander Killing ◽  
Induced Pluripotent

CRISPR/Cas9-mediated genome editing has demonstrated significant promise for genetic correction in autologous hematopoietic stem/progenitor cells (HSPCs) and induced pluripotent stem cells (iPSCs)

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Induced Pluripotent Stem Cells ◽  
Progenitor Cells ◽  
Genome Editing ◽  
Pluripotent Stem Cells ◽  
Genetic Disorders ◽  
Hematopoietic Stem ◽  
Hematological Disorders ◽  
Induced Pluripotent

According to current research, CRISPR/Cas9-mediated genome editing has shown enormous potential in the correction of genetic defects in autologous hematopoietic stem/progenitor cells (HSPCs) and induced pluripotent stem cells (iPSCs). Furthermore, the advancement of iPSC reprogramming technology as well as the CRISPR/Cas9 system has opened the door to new possibilities in the field of gene and cell therapy combinations. Despite the fact that there are a number of technological obstacles to overcome, CRISPR/Cas9 remains a promising therapeutic method with a great deal of potential for future gene therapy applications. Early success in treating hereditary hematological disorders opens the door to new options for treating other genetic disorders and constitutes a significant step forward in the development of gene therapy.

scholarly journals Small-scale screening of anticancer drugs acting specifically on neural stem/progenitor cells derived from human-induced pluripotent stem cells using a time-course cytotoxicity test

PeerJ ◽  
2018 ◽  
Vol 6 ◽  
pp. e4187 ◽  
Author(s):  
Hayato Fukusumi ◽  
Yukako Handa ◽  
Tomoko Shofuda ◽  
Yonehiro Kanemura
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Induced Pluripotent Stem Cells ◽  
Progenitor Cells ◽  
Anticancer Drugs ◽  
Pluripotent Stem Cells ◽  
Time Course ◽  
Neural Stem Progenitor Cells ◽  
Induced Pluripotent

Since the development of human-induced pluripotent stem cells (hiPSCs), various types of hiPSC-derived cells have been established for regenerative medicine and drug development. Neural stem/progenitor cells (NSPCs) derived from hiPSCs (hiPSC-NSPCs) have shown benefits for regenerative therapy of the central nervous system. However, owing to their intrinsic proliferative potential, therapies using transplanted hiPSC-NSPCs carry an inherent risk of undesired growth in vivo. Therefore, it is important to find cytotoxic drugs that can specifically target overproliferative transplanted hiPSC-NSPCs without damaging the intrinsic in vivo stem-cell system. Here, we examined the chemosensitivity of hiPSC-NSPCs and human neural tissue—derived NSPCs (hN-NSPCs) to the general anticancer drugs cisplatin, etoposide, mercaptopurine, and methotrexate. A time-course analysis of neurospheres in a microsphere array identified cisplatin and etoposide as fast-acting drugs, and mercaptopurine and methotrexate as slow-acting drugs. Notably, the slow-acting drugs were eventually cytotoxic to hiPSC-NSPCs but not to hN-NSPCs, a phenomenon not evident in the conventional endpoint assay on day 2 of treatment. Our results indicate that slow-acting drugs can distinguish hiPSC-NSPCs from hN-NSPCs and may provide an effective backup safety measure in stem-cell transplant therapies.

scholarly journals Generation of Induced Pluripotent Stem Cells and Neural Stem/Progenitor Cells from Newborns with Spina Bifida Aperta

2017 ◽  
Vol 11 (6) ◽  
pp. 870-879 ◽  
Author(s):  
Yohei Bamba ◽  
Masahiro Nonaka ◽  
Natsu Sasaki ◽  
Tomoko Shofuda ◽  
Daisuke Kanematsu ◽  
...  
Keyword(s):  
Stem Cells ◽  
Stem Cell ◽  
Spina Bifida ◽  
Induced Pluripotent Stem Cells ◽  
Progenitor Cells ◽  
Pluripotent Stem Cells ◽  
Clinical Use ◽  
Spina Bifida Aperta ◽  
Neural Stem Progenitor Cells ◽  
Induced Pluripotent

<sec><title>Study Design</title><p>We established induced pluripotent stem cells (iPSCs) and neural stem/progenitor cells (NSPCs) from three newborns with spina bifida aperta (SBa) using clinically practical methods.</p></sec><sec><title>Purpose</title><p>We aimed to develop stem cell lines derived from newborns with SBa for future therapeutic use.</p></sec><sec><title>Overview of Literature</title><p>SBa is a common congenital spinal cord abnormality that causes defects in neurological and urological functions. Stem cell transplantation therapies are predicted to provide beneficial effects for patients with SBa. However, the availability of appropriate cell sources is inadequate for clinical use because of their limited accessibility and expandability, as well as ethical issues.</p></sec><sec><title>Methods</title><p>Fibroblast cultures were established from small fragments of skin obtained from newborns with SBa during SBa repair surgery. The cultured cells were transfected with episomal plasmid vectors encoding reprogramming factors necessary for generating iPSCs. These cells were then differentiated into NSPCs by chemical compound treatment, and NSPCs were expanded using neurosphere technology.</p></sec><sec><title>Results</title><p>We successfully generated iPSC lines from the neonatal dermal fibroblasts of three newborns with SBa. We confirmed that these lines exhibited the characteristics of human pluripotent stem cells. We successfully generated NSPCs from all SBa newborn-derived iPSCs with a combination of neural induction and neurosphere technology.</p></sec><sec><title>Conclusions</title><p>We successfully generated iPSCs and iPSC-NSPCs from surgical samples obtained from newborns with SBa with the goal of future clinical use in patients with SBa.</p></sec>

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