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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

ABSTRACT: This SuperSeries is composed of the SubSeries listed below.

ORGANISM(S): synthetic construct Homo sapiens

PROVIDER: GSE275158 | 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 [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 [scRNA]

Project description: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%).

2024-08-21 | GSE275063 | 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

| PRJNA1160901 | ENA

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

| PRJNA1149701 | ENA

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]

| PRJNA1149703 | ENA

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]

| PRJNA1149034 | ENA

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: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]

| PRJNA1160904 | ENA

CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease.

Project description:Genome editing is potentially a curative technique available to all individuals with β-hemoglobinopathies, including sickle cell disease (SCD). Fetal hemoglobin (HbF) inhibits sickle hemoglobin (HbS) polymerization, and it is well described that naturally occurring hereditary persistence of HbF (HPFH) alleviates disease symptoms; therefore, reawakening of developmentally silenced HbF in adult red blood cells (RBCs) has long been of interest as a therapeutic strategy. Recent advances in genome editing platforms, particularly with the use of CRISPR-Cas9, have paved the way for efficient HbF induction through the creation of artificial HPFH mutations, editing of transcriptional HbF silencers, and modulating epigenetic intermediates that govern HbF expression. Clinical trials investigating BCL11A enhancer editing in patients with β-hemoglobinopathies have demonstrated promising results, although follow-up is short and the number of patients treated to date is low. While practical, economic, and clinical challenges of genome editing are well recognized by the scientific community, potential solutions to overcome these hurdles are in development. Here, we review the recent progress and obstacles yet to be overcome for the most effective and feasible HbF reactivation practice using CRISPR-Cas9 genome editing as a curative strategy for patients with SCD.

| S-EPMC8526756 | biostudies-literature

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