Associated Papers
Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides.
Fañanas-Baquero S et al.
Frontiers in Genome Editing, 2023
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.
Fañanas-Baquero S et al.
Molecular Therapy. Methods & Clinical Development, 2021
34485608 (PubMed )
Supplement: mmc1.pdf
Mutations in Kruppel-like factor 1 cause transfusion-dependent hemolytic anemia and persistence of embryonic globin gene expression.
Viprakasit V et al.
Blood, 2014
Select a paper to view details