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

Lignende værker

• Relative and Absolute Quantification of Aberrant and Normal Splice Variants in HBB(IVSI−110 (G > A)) β-Thalassemia
  af: Patsali, Petros, et al.
  Udgivet: (2020)
• Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies
  af: Loucari, Constantinos C., et al.
  Udgivet: (2018)
• 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
  af: Patsali, Petros, et al.
  Udgivet: (2019)
• Distinct miRNA Signatures and Networks Discern Fetal from Adult Erythroid Differentiation and Primary from Immortalized Erythroid Cells
  af: Papasavva, Panayiota L., et al.
  Udgivet: (2021)
• Genetic Modifiers at the Crossroads of Personalised Medicine for Haemoglobinopathies
  af: Stephanou, Coralea, et al.
  Udgivet: (2019)