Speakers: Maria Grazia Roncarolo, MD
Organizers:
Date: 1/27/2017
Time: 11:00-13:30
Registration fee (USD): ;
Location: Crowne Plaza Hotel, Foster City
Major Sponsor:
Vendor show vendors registered to date: (1)Thermo Fisher Scientific, SID
Registration: https://www.PBSS.org
Registration deadline:1/18/2017  (it will close sooner if the seating cap is reached)

About the Topic

Millions of people worldwide have serious genetic and acquired diseases for which there is no cure.Yet exciting advances in stem cell and gene therapy are creating opportunities to not only treat, but actually cure these diseases.The rate of scientific discovery has accelerated at such a pace that outcomes considered science fiction just ten years ago are now being seriously considered and pursued by experts around the globe. Genetic diseases of the blood, the immune system and the nervous system are now cured by inserting a normal copy of the relevant gene into somatic cells.

Primary immunodeficiencies (the bubble boys), were originally considered good candidates for gene therapy because they were well understood at the molecular level and because the target cells, the blood stem cells, are easily accessible and can be explanted, genetically corrected in the laboratory, and then retransplanted. Gene therapy with blood stem cells has been successfully developed as therapeutic strategy for several forms of primary immunodeficiency, including severe combined immunodeficiency (SCID)-X1, adenosine deaminase (ADA)-deficient SCID, and chronic granulomatous disease (CGD). Initial approaches were based on ex vivo retroviral vector mediated gene transfer of the therapeutic gene into the patient’s blood stem cells. The inclusion of reduced-dose conditioning in the ADA-SCID has allowed the engraftment of multipotent gene-corrected HSC at substantial level. Results of the ADA-SCID gene therapy trials have demonstrated safety, long-term restoration of immune competence and clinical benefit. Based on the data in the first 18 patients the product has been licensed to GSK that recently received European marketing authorization for Strimvelis.

The use of self-inactivating lentiviral vectors is providing significant advantages in gene regulation and reduction in the potential for adverse mutagenic events. Lentiviral vectors are currently used in clinical trials to cure SCID, Wiskott Aldrich syndrome and other primary immunodeficiencies but also blood disorders, such as thalassemia and sickle cell anemia, and metabolic diseases.

The prospects for continuing advancement of gene therapy to wider applications remain strong. Emerging methods for direct gene replacement by genome editing will broaden the use of gene therapy to other genetic and acquired diseases. In summary, in the near future the continuing advancement of stem cell and gene therapy will make it possible “to cure what is curable and make curable what is not”.