Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells

Ítems similars

• Short-hairpin RNA against aberrant HBB(IVSI-110(G>A)) mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells
  per: Patsali, Petros, et al.
  Publicat: (2018)
• Relative and Absolute Quantification of Aberrant and Normal Splice Variants in HBB(IVSI−110 (G > A)) β-Thalassemia
  per: Patsali, Petros, et al.
  Publicat: (2020)
• Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies
  per: Loucari, Constantinos C., et al.
  Publicat: (2018)
• The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements
  per: Patsali, Petros, et al.
  Publicat: (2019)
• Distinct miRNA Signatures and Networks Discern Fetal from Adult Erythroid Differentiation and Primary from Immortalized Erythroid Cells
  per: Papasavva, Panayiota L., et al.
  Publicat: (2021)