Session Details

Workshop 3 – Gene Replacement Therapy Using Adult Stem Cells
Friday, September 13, 2019 03:30 PM - 05:00 PM
Ballroom A
Patients with hemoglobinopathies including -thalassemia (-Thal) and Sickle Cell Disease (SCD) have chronic debilitating disease with chronic organ dysfunction and shortened life span. Both -Thal and SCD are autosomal recessive genetic disorders secondary to defects in the beta globin gene resulting in ineffective erythropoiesis and chronic hemolytic anemias, respectively. Historically, allogeneic stem cell transplantation has been the only therapeutic approach that has resulted in long term curative therapy. Due to the limited number of HLA matched and unmatched sibling donors, there has been a significant increase in the success of utilizing haploidentical unaffected family donors in the treatment of patients with high risk -Thal and SCD.
More recently, a number of novel approaches have been identified to enhance adult stem cell gene editing to correct genetic defects including the utilization of zinc-finger nucleases (ZFN), transcription activator-late effector nucleases (TALENs) and Cas9-gRNA nucleases (CRISPR-CAS9) and more recently, reversal of gamma globin silencing. Early studies with a variety of approaches of gene editing/correction of adult stem cells in patients with hemoglobinopathies have been demonstrated to be safe with some early signs of efficacy in a subset of patients.

Chair: Mitch Cairo, MD, New York Medical College, USA

Speakers:
Hematopoietic Stem Cell Transplantation and Gene Therapy for Hemoglobinopathies
Mitch Cairo, MD, New York Medical College, USA
Learning objectives:
•      To become familiar with the indications of allogeneic stem cell transplantation in patients with high risk hemoglobinopathies.
•      To understand the methods and outcomes of allogeneic stem cell transplantation in patients with high risk hemoglobinopathies.
•      To become acquainted with the safety and efficacy of gene therapy in patients with high risk hemoglobinopathies.

Gene Therapy for Fabry Disease Using Autologous CD34+ Hematopoietic Cells
Jeffrey Medin, PhD, Medical College of Wisconsin, USA
•      Understand how 'Metabolic Cooperativity' (aka Cross-Correction) drives outcomes in gene therapy for Fabry Disease.
•      Learn long-term clinical results in a lentivirus-mediated gene therapy trial that employed a non-myeloablative host conditioning regimen.
•      Evaluate biochemical and molecular biology results of this first-in-Fabry patients trial.

Hematopoietic Stem Cell Transplantation and Gene Therapy for Cerebral Adrenal Leukodystrophy (CALD)
Paul Orchard, MD, University of Minnesota – Pediatric BMT, USA
Learning objectives:
•      Describe the indications for cell based interventions for adrenoleukodystrophy (ALD)
•      Understand the rationale for and implications of newborn screening for ALD
•      Distinguish the pre-transplant factors important in determining outcomes in patients with cerebral ALD
•      Explain the differences in allogeneic and lentiviral based therapy for cerebal disease