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Description:
This session will describe new and established methods to modify DNA and their clinical application to treat hemoglobinopathies. New DNA editing techniques simplify the correction of mutations in human globin genes while the modification of induced pluripotent stem cells (iPSCs) and the use of zinc finger nucleases to increase the expression of the gamma-globin gene provide new opportunities for curative therapy for sickle cell disease and thalassemia. Gene therapy with lentiviral vectors is being evaluated in clinical trials to correct mutations in the gamma-globin gene.
Dr. Mali will describe systems for gene editing including zinc finger nucleases, transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 endonuclease. He will also discuss the requirements for efficacy of genome targeting to enable therapeutic applications.
Dr. Townes will present the development and pre-clinical evaluation of patient-specific iPSCs and CRISPR/Cas gene correction for the treatment of hemoglobinopathies.
Dr. Leboulch will describe the preclinical development and clinical application of lentiviral vectors for sickle cell disease and thalassemia.