Presentation
Resources & publications
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Journal (source)Prog Mol Biol Transl SciGenome editing approaches to β-hemoglobinopathies.
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2022Journal (source)Nat Commun
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the...
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2023Journal (source)Mol Ther Nucleic Acids
Novel lentiviral vectors for gene therapy of sickle cell disease combining ge...
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2022Journal (source)Stem Cells Transl Med
CRISPRthripsis: The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy.
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2022Journal (source)Mol Ther
Combination of lentiviral and genome editing technologies for the treatment o...
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2021Journal (source)Front Genome Ed
Base and Prime Editing Technologies for Blood Disorders.