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Gene therapy using genome-edited human iPS cell-derived neural stem cells overcomes refractory course of invasive glioma stem cells

ABSTRACT: Gene therapy using genome-edited human iPS cell-derived neural stem cells overcomes refractory course of invasive glioma stem cells

PROVIDER: PRJNA632518 | ENA |

REPOSITORIES: ENA

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			Action	DRS
	SRR11783525_1.fastq.gz	Fastqsanger.gz
	SRR11783525_2.fastq.gz	Fastqsanger.gz
	SRR11783526_1.fastq.gz	Fastqsanger.gz
	SRR11783526_2.fastq.gz	Fastqsanger.gz
	SRR11783527_1.fastq.gz	Fastqsanger.gz

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Gene therapy using genome-edited human iPS cell-derived neural stem cells overcomes refractory course of invasive glioma stem cells

Project description:Spatiotemporal analyses using brain slice culture and brain clearing demonstrated that human induced pluripotent stem cell-derived neural stem cells (iPSC-NSCs) possess higher tumor-trophic migratory capacity than fetal NSCs, adipose tissue and bone marrow derived mesenchymal stem cells (MSCs). NSCs expressing prodrug converting enzyme fusion gene exhibited strong anti-tumor effect for glioma stem cell in vivo models. The present research concepts may become a platform of cell-based gene therapy for glioma.

2022-09-16 | GSE150470 | GEO

CRISPR-edited human ES-derived oligodendrocyte progenitor cells improve remyelination in rodents

Project description:In Multiple Sclerosis (MS), inflammatory demyelinated lesions in the brain and spinal cord lead to neurodegeneration and progressive disability. Remyelination can restore fast saltatory conduction and neuroprotection but is inefficient in MS especially with increasing age, and is not yet improvable with therapies. Intrinsic and extrinsic inhibition of oligodendrocyte progenitor cell (OPC) function contributes to remyelination failure, and we hypothesised that the transplantation of ‘improved’ OPCs, genetically edited to overcome these obstacles, could improve remyelination. Here, we edited human(h) embryonic stem cell-derived OPCs to be unresponsive to a chemorepellent released from chronic MS lesions, and transplanted them into rodent models of chronic lesions. Edited hOPCs displayed enhanced migration and remyelination compared to controls, regardless of the host age and length of time post-transplant. This study demonstrates that genetic manipulation and transplantation of hOPCs overcomes the negative environment inhibiting remyelination, with translational implications for therapeutic strategies for people with progressive MS.

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Transcriptomic analysis of edited human hematopoietic stem/progenitor cells

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2022-09-06 | GSE191135 | GEO

Transcriptomic analysis of edited human hematopoietic stem/progenitor cells

Project description:Transcriptomic analysis of edited human hematopoietic stem/progenitor cells

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Controlling Genetic Heterogeneity in Gene-edited Hematopoietic Stem Cells by Single Cell Expansion

Project description:Gene editing using engineered nucleases frequently produces unintended genetic lesions in hematopoietic stem cells (HSCs). Gene-edited HSC cultures thus contain heterogenous populations, the majority of which either do not carry the desired edit or harbor unwanted mutations. In consequence, transplanting edited HSCs carries the risks of suboptimal efficiency and of unwanted mutations in the graft. Here, we present an approach for expanding gene-edited HSCs at clonal density, allowing for genetic profiling of individual clones before transplantation. We achieved this by developing a defined, polymer-based expansion system and identifying long-term expanding clones within the CD201+CD150+CD48-c-Kit+Sca-1+Lin-(KSL) population of pre-cultured HSCs. This dataset compares the gene expression in three different populations: (1) CD201+CD150+CD48-KSL (2) CD201+CD150+CD48+KSL and (3) CD201-KSL cells.

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Project description:This phase I trial studies the side effects and best dose of monoclonal antibody therapy before stem cell transplant in treating patients with relapsed or refractory lymphoid malignancies. Radiolabeled monoclonal antibodies, such as yttrium-90 anti-CD45 monoclonal antibody BC8, can find cancer cells and carry cancer-killing substances to them without harming normal cells. When the healthy stem cells from a donor are infused into the patient they may help the patient’s bone marrow make stem cells, red blood cells, white blood cells, and platelets. Giving radiolabeled monoclonal antibody before a stem cell transplant may be an effective treatment for relapsed or refractory lymphoid malignancies.

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Effects on gene expression of doxorubicin treatment in genome-edited human induced pluripotent stem cell-derived cardiomyocytes

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Project description:Refractory Cancer (RC) Program

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