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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.

2023-08-23 | GSE241451 | GEO

Transcriptomic analysis of edited human hematopoietic stem/progenitor cells

Project description:We have developed a therapeutic strategy for beta-hemoglobinopathies aimed at reactivating fetal hemoglobin expression in red blood cells derived from human hematopoietic stem/progenitor cells edited with CRISPR/Cas9 nucleases, cytidine or adenine base editors targeting the fetal gamma-globin promoters. Here, we report the transcriptomic changes occurring in human hematopoietic stem/progenitor cells (obtained from healthy donors) 48 h after transfection with CRISPR/Cas9 nucleases, cytidine or adenine base editors.

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

| PRJNA789853 | ENA

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.

2023-07-19 | GSE232527 | GEO

Monoclonal Antibody Therapy Before Stem Cell Transplant in Treating Patients With Relapsed or Refractory Lymphoid Malignancies

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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The reparative immunologic consequences of stem cell transplantation as a cellular therapy for refractory Crohn’s disease

Project description:Background Treatment strategies for Crohn’s disease (CD) suppress diverse inflammatory pathways but many patients remain refractory to treatment. Autologous hematopoietic stem cell transplantation (SCT) is an emerging therapy for medically-refractory CD though the mechanisms through which it circumvents refractory pathophysiology are unknown. Objective The objective of this study is to understand how the immune system reconstitutes post-SCT and whether SCT may function as a cellular therapy restoring appropriately responsive immune cell populations from hematopoietic stem cells (HSCs). Design Adults with CD with active clinical and endoscopic disease who failed available medical therapies were enrolled in a Phase II study of SCT for refractory CD (n=19). Blood and intestinal samples were collected longitudinally and analyzed using CyTOF, scRNA-seq and TCRβ-seq. Stem cell autografts were functionally assayed in mouse xenograft models. Results scRNA-seq and CyTOF analyses reveal that SCT predominantly affected the intestinal myeloid lineage with loss of inflammatory populations and return of macrophages capable of supporting mucosal healing. Xenograft models using patient HSCs suggested HSCs support the early reconstitution of the myeloid lineage and reveal an impairment of short and long-term HSC engraftment that may determine SCT outcomes. Conclusions This study suggests SCT functions as a myeloid-directed cellular therapy reinforcing the critical role of macrophages in refractory CD pathophysiology and as a target for cellular therapies. Furthermore, we report an unrecognized functional heterogeneity among HSC subpopulations in CD that may be relevant to our understanding of CD treatment and pathophysiology.

2025-01-23 | GSE284677 | GEO

Neuroprotective Effects of Genome-Edited Human iPS Cell-Derived Neural Stem/Progenitor Cells on Traumatic Brain Injury.

Project description:Despite developing neurosurgical procedures, few treatment options have achieved functional recovery from traumatic brain injury (TBI). Neural stem/progenitor cells (NS/PCs) may produce a long-term effect on neurological recovery. Although induced pluripotent stem cells (iPSCs) can overcome ethical and practical issues of human embryonic or fetal-derived tissues in clinical applications, the tumorigenicity of iPSC-derived populations remains an obstacle to their safe use in regenerative medicine. We herein established a novel treatment strategy for TBI using iPSCs expressing the enzyme-prodrug gene yeast cytosine deaminase-uracil phosphoribosyl transferase (yCD-UPRT). NS/PCs derived from human iPSCs displayed stable and high transgene expression of yCD-UPRT following CRISPR/Cas9-mediated genome editing. In vivo bioluminescent imaging and histopathological analysis demonstrated that NS/PCs concentrated around the damaged cortex of the TBI mouse model. During the subacute phase, performances in both beam walking test and accelerating rotarod test were significantly improved in the treatment group transplanted with genome-edited iPSC-derived NS/PCs compared with the control group. The injury area visualized by extravasation of Evans blue was smaller in the treatment group compared with the control group, suggesting the prevention of secondary brain injury. During the chronic phase, cerebral atrophy and ventricle enlargement were significantly less evident in the treatment group. Furthermore, after 5-fluorocytosine (5-FC) administration, 5-fluorouracil converted from 5-FC selectively eliminated undifferentiated NS/PCs while preserving the adjacent neuronal structures. NS/PCs expressing yCD-UPRT can be applied for safe regenerative medicine without the concern for tumorigenesis.

| S-EPMC10267696 | biostudies-literature

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

Project description:To gain further insight into the mechanisms of doxorubicin-induced cardiotoxicty in genome-edited isogenic induced pluripotent stem cell-derived cardiomyocytes with or without the RARG variant S427L, we performed RNA-seq in doxorubicin- or vehicle-treated cardiomyocytes in both genotypes and investigate the differential expression genes as well as enriched molecular pathways.

2022-04-13 | GSE181517 | GEO

Refractory Cancer (RC) Program

Project description:Refractory Cancer (RC) Program

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