Gene Therapy for Hemophilia, Blindness, and Cancer, and the Tools That Make it Possible at the HMGS 2014 Symposium

Every blogger has those times when life gets busy and their blog takes a back seat.  For me, this summer has been one of those times.  Between meetings, research, our family trip back home, and the general effort involved in being a scientist, I have been behind in updating this blog.  Despite my blog slacking, I'm sure it will be worth it when I have more cool stuff to write about in the next couple of months (especially when I have cool new research findings to talk about!).  So let's get started with some awesome catchup!

Dr. Maus presenting her research to the students attending
the symposium.

A month ago, we here at Penn hosted the annual Howard Hughes Medical Institute (HHMI) Med Into Grad Scholars (HMGS) symposium for the participating northeast schools.  This program was made to promote translational research in PhD training by integrating more medically relevant training into the PhD candidate curriculum (including coursework and clinical clerkships).  For more information about the program, and to read about the symposium last year, check out my post here.


The topic for this year's symposium was Gene Therapy, which made sense because we have a lot of great gene therapy researchers here at Penn who were able to give some talks.  These presenters included Dr. Katharine High, Dr. Jean Bennett, Dr. Jim Wilson, and Dr. Marcela Maus (faculty profiles in the abstract book).  Dr. High talked about her research toward using gene therapeutic approaches to treat hemophilia, which has come a long way over the years and is yielding great results.  Dr. Bennett discussed her work in developing gene therapies for treating congenital blindness, which is just amazing!  Because of this research, there are actually children who were blind but are now able to see after being treated with this gene therapy.  Check out the video clip below to see the remarkable results, and to get some more information.

Dr. Wilson, another leader in the gene therapy field, talked about his important work in developing the technologies and tools used in gene therapy.  Primarily these tools have been viruses, and more specifically Dr. Wilson discussed his work with the Adeno-Associated Virus (AAV).  Finally we concluded the evening with Dr. Maus discussing her work in using Chimeric Antigen Receptor (CAR) T-cells to treat cancer. These are treatments which involve engineering T-cells (immune cells your body uses to kill other dangerous cells) to kill cancer cells, and this approach has been shown to be incredibly successful in treating certain types of cancer (see here for a general NY Times article about related research).

The following day we had some awesome student talks, as well as a great poster session where more students presented and discussed their research.  This was a great time to meet with other students with similar interests in translational research, discuss their science, and learn about their experiences.  Following these events we concluded the symposium with lunch, after which everybody got on their planes, trains, and automobiles to head home (and hopefully had a smoother trip than Steve Martin).

Students discussing their research posters the morning
of the second day.

Unfortunately this year also marked the conclusion of the HHMI funding cycle for this Med Into Grad Program.  Although this cessation of funding is a bummer, it is not unexpected because the purpose of this program was for the HHMI to stimulate an interest in the individual schools providing their own programs after the HHMI money ran out.  Fortunately this has been successful, and the University of Pennsylvania is excited to continue this program.  It also sounds like most of the other schools of the northeast region will continue their own programs, and hopefully we can even continue to have this annual meeting.

For more information about the symposium, or to read the abstract books and faculty speaker profiles, check out this website my colleague and I put together for the symposium.  We are also going to be restructuring the overall program website in the near future, so there is a chance this link will break.  If I forget to update the link, drop me a reminder in the comments below and I will be sure to fix it up.  We also had a lot of pictures from the symposium (I included a couple above) and we will be getting those all up on our website soon, so keep an eye out for those.