IDEAS home Printed from https://ideas.repec.org/a/nat/natcom/v7y2016i1d10.1038_ncomms13173.html
   My bibliography  Save this article

Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy

Author

Listed:
  • Jennifer E. Adair

    (Fred Hutchinson Cancer Research Center
    University of Washington, 1410 Campus Parkway, Seattle, Washington 98195, USA)

  • Timothy Waters

    (Miltenyi Biotec Inc.)

  • Kevin G. Haworth

    (Fred Hutchinson Cancer Research Center)

  • Sara P. Kubek

    (Fred Hutchinson Cancer Research Center)

  • Grant D. Trobridge

    (Washington State University Spokane)

  • Jonah D. Hocum

    (Washington State University Spokane)

  • Shelly Heimfeld

    (Fred Hutchinson Cancer Research Center)

  • Hans-Peter Kiem

    (Fred Hutchinson Cancer Research Center
    University of Washington, 1410 Campus Parkway, Seattle, Washington 98195, USA)

Abstract

Haematopoietic stem cell (HSC) gene therapy has demonstrated potential to treat many diseases. However, current state of the art requires sophisticated ex vivo gene transfer in a dedicated Good Manufacturing Practices facility, limiting availability. An automated process would improve the availability and standardized manufacture of HSC gene therapy. Here, we develop a novel program for semi-automated cell isolation and culture equipment to permit complete benchtop generation of gene-modified CD34+ blood cell products for transplantation. These cell products meet current manufacturing quality standards for both mobilized leukapheresis and bone marrow, and reconstitute human haematopoiesis in immunocompromised mice. Importantly, nonhuman primate autologous gene-modified CD34+ cell products are capable of stable, polyclonal multilineage reconstitution with follow-up of more than 1 year. These data demonstrate proof of concept for point-of-care delivery of HSC gene therapy. Given the many target diseases for gene therapy, there is enormous potential for this approach to treat patients on a global scale.

Suggested Citation

  • Jennifer E. Adair & Timothy Waters & Kevin G. Haworth & Sara P. Kubek & Grant D. Trobridge & Jonah D. Hocum & Shelly Heimfeld & Hans-Peter Kiem, 2016. "Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy," Nature Communications, Nature, vol. 7(1), pages 1-10, December.
    • Handle: RePEc:nat:natcom:v:7:y:2016:i:1:d:10.1038_ncomms13173
    DOI: 10.1038/ncomms13173
    as

    Download full text from publisher

    File URL: https://www.nature.com/articles/ncomms13173
    File Function: Abstract
    Download Restriction: no
    File URL: https://libkey.io/10.1038/ncomms13173?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    More about this item

    Statistics

    Access and download statistics

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:nat:natcom:v:7:y:2016:i:1:d:10.1038_ncomms13173. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    We have no bibliographic references for this item. You can help adding them by using this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Sonal Shukla or Springer Nature Abstracting and Indexing (email available below). General contact details of provider: http://www.nature.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.