JAN 09, 2020 10:00 AM PST
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Engineering the hematopoietic system to treat non-hematological disorders

Speakers
  • Assistant Professor of Pediatrics (Genetics) and of Pediatrics (Stem Cell Transplantation), Stanford University
    Biography
      Dr. Gomez-Ospina is currently an Assistant Professor in the Department of Pediatrics. For her clinical practice she sees patients with suspected genetic disorders and is also in charge of the enzyme replacement service for lysosomal storage disorders at Lucile Packard Children's hospital. Despite, her young career, she has been the lead author in research studies in the New England Journal of Medicine, Cell, Nature Communications, and American Journal of Medical Genetics. Dr. Gomez-Ospina has championed the idea of commandeering the hematopoietic system to express proteins needed in other organs, including the brain. She established an adaptable platform for the treatment of lysosomal enzyme deficiencies and performed a first-of-its-kind preclinical study to support the clinical development of autologous transplantation of genome edited cells to treat patients with Mucopolysaccharidosis type I (Hurler syndrome). Beyond delivering lysosomal enzymes, this platform has potential implications for delivering many kinds of therapeutic proteins to the brain. In addition to therapy development, Dr. Gomez-Ospina led the discovery and characterization of a new infantile cholestatis syndrome caused by mutations in the bile acid receptor and, as part of large multi-institutional collaborations, participated in the discovery of multiple intellectual disability syndromes.

    Abstract:
    DATE:  January 9, 2020
    TIME:   10:00am PST, 1:00pm EST
     
    Genome editing, a mechanism to rewrite DNA sequences, has the potential to create a new generation of precise genetic therapies that can ameliorate or cure genetic disorders. For genome editing to be therapeutic, it must not only produce the desired genetic modifications, but also efficiently target the appropriate cells in the appropriate organs. Ex vivo and in vivo delivery approaches are the main platforms used to achieve modification of target tissues. While ex vivo modification of hematopoietic stem cells is being primarily developed to target genetic diseases of the blood such as hemoglobinopathies and immune deficiencies, it also has great potential to treat non-hematological multi-systemic diseases. In this webinar we will discuss how the hematopoietic system can be engineered using genome editing to produce, secrete, and deliver therapeutic proteins to multiple organs, including the central nervous system. Specifically, we discuss recent data, and provide specific examples, regarding how such an approach can become a therapeutic platform for a large group of genetic diseases caused by deficiencies in lysosomal enzymes. We also review a CRISPR/Cas9-based, good-manufacturing-practice-compatible protocol for modification of hematopoietic stem cells ex vivo. 
     
    Learning Objectives:
    • Review the use sgRNA/Cas9 ribonucleoprotein (RNP) and adeno-associated viral vector serotype six (AAV6) delivery of the homologous templates for genome editing of human hematopoietic stem and progenitor cells.
    • Review proof-of-concept studies showing that human genome edited hematopoietic stem cells can correct disease manifestations in multi-systemic, non-hematological diseases.
    • Discuss broader applications of this technology as a mean to deliver therapeutic proteins to multiple organ systems, including the brain.
     
    Webinars will be available for unlimited on-demand viewing after live event.
     
    LabRoots is approved as a provider of continuing education programs in the clinical laboratory sciences by the ASCLS P.A.C.E. ® Program. By attending this webinar, you can earn 1 Continuing Education credit once you have viewed the webinar in its entirety.
     
     
     

     


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