JAN 09, 2020 10:00 AM PST

Engineering the hematopoietic system to treat non-hematological disorders

Sponsored by: STEMCELL Technologies
C.E. Credits: P.A.C.E. CE Florida CE
Speaker
  • 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.
     
     
     

     


    Show Resources
    You May Also Like
    MAY 11, 2021 10:00 AM PDT
    C.E. CREDITS
    MAY 11, 2021 10:00 AM PDT
    Date: May 11, 2021 Time: 10:00zm PDT Your samples are some of the most valuable assets in the laboratory. After spending countless hours on extraction and preparation, your conclusions could...
    JUN 09, 2021 7:00 AM PDT
    Add to Calendar Select one of the following: iCal Google Calendar Outlook Calendar Yahoo Calendar
    C.E. CREDITS
    JUN 09, 2021 7:00 AM PDT
    Add to Calendar Select one of the following: iCal Google Calendar Outlook Calendar Yahoo Calendar
    Date: June 9, 2021 Time: 09 June 2021, 7am PDT, 10am EDT, 4pm CEST cells with dramatic implications on the validity of past cell culture related research. The fact that at least 509 cell lin...
    DEC 02, 2020 8:00 AM PST
    C.E. CREDITS
    DEC 02, 2020 8:00 AM PST
    DATE: December 2nd, 2020 TIME: 08:00am PDT, 11:00pm EDT Bioreactors and shakers are used to cultivate microorganisms, plant, insect, and mammalian cells in different volumes. Upscaling of pr...
    NOV 16, 2020 8:00 AM PST
    C.E. CREDITS
    NOV 16, 2020 8:00 AM PST
    Date: November 16, 2020 Time: 8:00am (PST), 11:00am (EST) CRISPR screening has become the prime discovery tool in modern biomedical research and drug discovery. At the same time, most screen...
    DEC 15, 2020 10:00 AM PST
    C.E. CREDITS
    DEC 15, 2020 10:00 AM PST
    DATE: December 15, 2020 TIME: 10:00am PST Scientists from Thermo Fisher Scientific will walk us through the world of microorganisms. They will discuss their most recent research on viruses,...
    MAR 18, 2021 8:00 AM PDT
    C.E. CREDITS
    MAR 18, 2021 8:00 AM PDT
    DATE: March 18, 2021 TIME: 8:00am PDT Sequencing of bulk cells, single cells, and nuclei is opening doors in the understanding of complex biological processes....
    JAN 09, 2020 10:00 AM PST

    Engineering the hematopoietic system to treat non-hematological disorders

    Sponsored by: STEMCELL Technologies
    C.E. Credits: P.A.C.E. CE Florida CE

    Specialty

    Molecular Biology

    Cell Biology

    Immunology

    Cancer Research

    Biotechnology

    Laboratory Testing

    Genetics

    Molecular Diagnostics

    Cell Culture

    Clinical Diagnostics

    Microbiology

    Gene Expression

    Infectious Disease

    Clinical Research

    Cancer Therapeutics

    Geography

    North America62%

    Europe16%

    Asia14%

    South America3%

    Africa2%

    Oceania1%

    Registration Source

    Website Visitors36%

    Email Promotions28%

    Client/Sponsor Promotions25%

    WAVE Newsletter Promotion8%

    Trending Newsletter Promotion2%

    Other1%

    Job Title

    Medical Laboratory Technician15%

    Educator/Faculty10%

    Executive10%

    Research Scientist10%

    Student9%

    Lab Management8%

    Clinical Laboratory Scientist8%

    Scientist8%

    Facility/Department Manager7%

    Medical Doctor/Specialist5%

    Post Doc4%

    Biologist2%

    QC/QA2%

    Pathologist1%

    Organization

    Academic Institution25%

    Hospital17%

    Clinical Laboratory10%

    Biotech Company9%

    Research Institute5%

    Medical Center3%

    Government3%

    Pharmaceutical Company2%

    Medical School2%

    Government/public2%

    Non-Profit Organization2%

    Independent Laboratories1%

    Consultant1%

    Life Science Company1%

    Other3%


    Show Resources
    Loading Comments...
    Show Resources
    Attendees
    • See more