Publications by authors named "Michelle Mojadidi"
We examined the efficiency, specificity, and mutational signatures of zinc finger nucleases (ZFNs), transcriptional activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 systems designed to target the gene encoding the transcriptional repressor BCL11A, in human K562 cells and human CD34 progenitor cells. ZFNs and TALENs were delivered as in vitro transcribed mRNA through electroporation; CRISPR/Cas9 was codelivered by Cas9 mRNA with plasmid-encoded guideRNA (gRNA) (pU6.g1) or in vitro transcribed gRNA (gR.
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- Targeted genome editing technologies, including TALENs and CRISPR/Cas9, can effectively correct the sickle cell disease mutation in the β-globin gene within hematopoietic stem cells.
- The correction allows for the production of red blood cells that produce normal hemoglobin proteins instead of the abnormal ones associated with sickle cell disease.
- In experiments with patient-derived CD34+ cells, CRISPR/Cas9 led to over 18% gene modification and successfully corrected the mutation, demonstrating its potential for treating this genetic disorder.
Purpose: Angiogenesis is essential for physiological processes as well as for carcinogenesis. New approaches to cancer therapy include targeting angiogenesis. One target is VEGF-A and its receptor VEGFR2.
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