This is the time to finalize plans to attend the 2019 annual meeting in Tampa, Florida. The scientific program is complete and the social activities are scheduled to facilitate meeting with old friends and provide opportunities to build new relationships. We are very fortunate to have Professor June Medford as our Keynote Speaker. She will address “Synthetic Biology for Engineering Plant Genetic Circuits: from Predictable Electronic-like Functions to Innovative Desalination.” This topic should be of interest to all society members. One of the great strengths of the SIVB annual meeting is the opportunity to view topics in biology from outside the confines of our own research experience. The program committee has done a particularly good job in preparing a very diverse program for the meeting. The Tampa Marriott Water Street will be a unique venue for the meeting. It is located on the water with good meeting rooms and areas for our social activities. The Tuesday evening event will be at the Florida Aquarium which is not far from the hotel. These events are always fully subscribed so if you have an interest in attending, please purchase your ticket through the Society’s website before the meeting. There are a limited number of reduced price tickets for students.

The SIVB has had a longstanding interest in genetic modification of cells for the introduction of desired traits, selective modification of metabolic pathways or mitigation of genetically-based disease (gene therapy). Heritable genetic modification of the whole organism has been more common in the plant kingdom but not without some controversy. On the animal side, the production of genetically modified research animals or cells has been very successful but successful gene therapy in humans has been much more of a challenge. The promise of human gene therapy seemed bright twenty years ago but notable failures have made the public and scientific community much more cautious. Recently some very good news has come forward in human gene therapy. Severe Combined Immunodeficiency Disease-X1 (SCID-X1) is a condition where the patient fails to produce T-cells, B-cells and natural killer cells and thus has no functioning cellular or humoral immunity. It is the result of a mutation in the gene (IL2RG) that encodes for the γ-chain shared by multiple cytokine receptors required for development of the immune cells. Fortunately, this is a rare condition and some patients can be treated by a bone marrow transplant from am HLA-matched normal sibling. For those patients without a matched normal sibling, the prospects have been dire. Extensive immunosuppression had to be used to prevent bone marrow rejection and success was by no means assured. Even early gene therapy approaches had their own negative consequences such as the induction of leukemia. A very recent publication reports of a collaboration between St. Jude Children’s Research Hospital and University of California, San Francisco which used a specially designed Lentiviral vector to transduce allogeneic bone marrow progenitor cells in vitro with a functional IL2RG gene sequence[1]. A buffer sequence was added to prevent deleterious activation of downstream genes at the insertion site associated with induction of leukemia. Low-dose Busulfan was given to encourage colonization of the recipient bone marrow. The results of eight patients were presented and the results are most impressive. Restoration of the immune function (T cells, B cells and natural killer cells) was clear as was the absence of deleterious side effects. The median age of the patients was 3.5 months and the median post-treatment follow up was 16.4 months (range 6 to 24 months). To me, there were several particularly notable aspects to this study: the number of individuals and centers that collaborated to bring all the tools together; the care in addressing the known limitation of previous gene therapy protocols; and the willingness to follow the patients for a relatively long period before publication of the work. This work can be counted as a success for gene therapy and a model for addressing other disease states.

I look forward to seeing as many people as possible in Tampa.

Respectfully yours,

John

John W. Harbell, Ph.D.
President
Society for In Vitro Biology

[1] Ewelina Mamcarz et al, Lentivirus gene therapy combined with low-dose Busulfan in infants with SCID-X1. New England Journal of Medicine 380:1525-1534, 2019.