Sort   by:  
 Page size 
UnknownCRISPR editing to mimic porphyria combined with light: A new preclinical approach for prostate cancer.
Thanks to its very high genome-editing efficiency, CRISPR-Cas9 technology could be a promising anticancer weapon. Clinical trials using CRISPR-Cas9 nuclease to ex vivo edit and alter immune cells are ongoing. However, to date, this strategy still has not been applied in clinical practice to d...
S-EPMC10899051 | biostudies-literature
UnknownPreventing Pluripotent Cell Teratoma in Regenerative Medicine Applied to Hematology Disorders.
Iatrogenic tumorigenesis is a major limitation for the use of human induced pluripotent stem cells (hiPSCs) in hematology. The teratoma risk comes from the persistence of hiPSCs in differentiated cell populations. Our goal was to evaluate the best system to purge residual hiPSCs before graft without...
S-EPMC5442801 | biostudies-literature
UnknownMutation-Specific Guide RNA for Compound Heterozygous Porphyria On-target Scarless Correction by CRISPR/Cas9 in Stem Cells.
CRISPR/Cas9 is a promising technology for gene correction. However, the edition is often biallelic, and uncontrolled small insertions and deletions (indels) concomitant to precise correction are created. Mutation-specific guide RNAs were recently tested to correct dominant inherited diseases, sparin...
S-EPMC7486222 | biostudies-literature
UnknownCRISPR-Cas9 globin editing can induce megabase-scale copy-neutral losses of heterozygosity in hematopoietic cells.
CRISPR-Cas9 is a promising technology for gene therapy. However, the ON-target genotoxicity of CRISPR-Cas9 nuclease due to DNA double-strand breaks has received little attention and is probably underestimated. Here we report that genome editing targeting globin genes induces megabase-scale losses of...
S-EPMC8363739 | biostudies-literature
UnknownCRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations.
CRISPR-Cas9 is a promising technology for genome editing. Here we use Cas9 nuclease-induced double-strand break DNA (DSB) at the UROS locus to model and correct congenital erythropoietic porphyria. We demonstrate that homology-directed repair is rare compared with NHEJ pathway leading to on-target i...
S-EPMC6408493 | biostudies-literature
TranscriptomicsHIF knockdowns in human hematopoietic stem cells
Hematopoietic stem cells (HSCs), which reside in bone marrow niches, are exposed to low levels of oxygen and follow an oxygen gradient throughout their differentiation. Hypoxia-inducible factors (HIFs) are the main factors regulating the cell response to oxygen variation. Recent studies using condit...
ORGANISM(S): Homo sapiens 
2014-01-01 | E-GEOD-49897 | biostudies-arrayexpress
Sort   by:  
 Page size