CRISPR gene therapy shows promise against blood diseases.

  title={CRISPR gene therapy shows promise against blood diseases.},
  author={Heidi Ledford},
CRISPR enriches for cells with mutations in a p53-related interactome, and this can be inhibited
It is shown that cells deficient in p53, as well as in genes of a core CRISPR-p53 tumor suppressor interactome, are enriched in a cell population whenCRISPR is applied, and that transient p53 inhibition suppresses the enrichment of cells with these mutations.
CRISPR/Cas9-Induced DNA Damage Enriches for Mutations in a p53-Linked Interactome: Implications for CRISPR-Based Therapies
It is shown thatCRISPR/Cas9-induced double-stranded DNA breaks enrich for cells deficient in p53 and in genes of a core CRISPR–p53 tumor suppressor interactome, which can be suppressed by transient inhibition of p53.


CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
Electroporation of CD34+ hematopoietic stem and progenitor cells obtained from healthy donors was performed, with CRISPR-Cas9 targeting the BCL11A erythroid-specific enhancer, and approximately 80% of the alleles at this locus were modified, with no evidence of off-target editing.
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.
This study validates BCL11A inhibition as an effective target for HbF induction and provides preliminary evidence that shmiR-based gene knockdown offers a favorable risk-benefit profile in sickle cell disease.
Sickle cell anemia a molecular disease.
The erythrocytes of certain individuals possess the capacity to undergo reversible changes in shape in response to changes in the partial pressure of oxygen, and these cells change their forms from the normal biconcave disk to crescent, holly wreath, and other forms.