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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease [scRNA]

ABSTRACT: To determine the heterogeneity of fetal hemoglobin induction at a single-cell level, CD235a+ erythroid cells were extracted from mouse BM after 17 weeks xenotransplantation. Single-cell RNA-sequencing (10x Genomics) showed 82% of the cells more than 30% g-globin expression in edited cells compared to unedited controls (26.7%).

ORGANISM(S): Homo sapiens

PROVIDER: GSE275063 | GEO | 2024/08/21

REPOSITORIES: GEO

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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

Project description:To determine the genome-wide spectrum and persistence of genomic rearrangements, we applied UniDirectional Targeted Sequencing (UDiTaS) in edited normal donor CD34+ HSPCs. At day 5 and 14 post-electroporation, no reproducible translocations or structural genomic re-arrangements were identified.

2024-09-14 | GSE277175 | GEO

Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease [sgRNA]

Project description:To identify potential single-guide RNA (sgRNAs) contaminants in the GMP HBG-sgRNA, we performed SMARTer smRNA-sequencing (Takara). 80.79% of the 5' spacer sequence perfectly matched the targeting sgRNA sequence and no evidence of contaminants sgRNAs that could target other genomic regions.

2024-08-21 | GSE275157 | GEO

Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease [CHANGE-seq]

Project description:To define the genome-wide activity of Cas9-sgRNA directed at the gamma-globin promoter genes, we performed CHANGE-seq and identified 678 off-target sites from plerixafor-mobilized normal donors of African American ancestry (pre-GMP engineering runs, n = 3). Most of these off-targets are in intergenic and intronic regions of the human genome, as expected based on their relative genomic proportions. We selected the 277 CHANGE-seq candidate sites reproducibly identified in 2 or more biological donors and in silico predicted sites for multiplexed targeted amplicon sequencing (rhAmp-seq, IDT).

2024-09-14 | GSE277176 | GEO

Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

Project description:Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

Project description:Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease [sgRNA]

Project description:Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease [sgRNA]

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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease [scRNA]

Project description:Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease [scRNA]

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Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease [CHANGE-seq]

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RNA-seq of RS4:11 cell line populations that are unedited (SPI1 +/+), mono (SPI1 +/-) and biallelicaly edited (SPI1 -/-)

Project description:The goal of the experiment was to identify which genes are differentially expressed between the unedited and SPI1-edited populations. The RS4:11 cell line was edited both mono and biallelicaly via electroporation with guides and Cas9. Following editing, RNA from unedited (SPI1 +/+), mono (SPI1 +/-) and biallellicaly edited (SPI1 -/-) cells were extracted through the Direct-zol RNA Microprep kit. cDNA libraries for sequencing were then prepared using the TruSeq Stranded mRNA Library Prep Kit and the IDT for Illumina-TruSeq RNA UD Indexes (Illumina). Samples were then sequenced on the Illumina NovaSeq platform.

2021-03-24 | E-MTAB-8677 | biostudies-arrayexpress

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