A lot of times when these patients are first diagnosed with these rare
inherited neurologic diseases their reality as they know it ceases to exist.
These parents have to suddenly become experts in a disease they've never
heard of. T he parents turn to the scientific community. What is known about this disease?
Is there a clinical trial? Is there anything that I can offer my kid and that's really where the
researchers step in. Are we able to understand what is causing a certain disease and can we
develop a therapy that would really change the outcome of these kids lives. We offer them hope.
One of the strategic goals of this institution is to really learn from every child and to really
use our research to change the lives of children our mission in the center for gene therapy is to
really dovetail to be really part of that strategic mission of the larger institution
to learn from all of the patients who come through our doors and in turn
to accelerate into the clinic therapies that will have a meaningful impact on their lives
and by extension lives of all children with health issues with genetic disorders that we
can treat by these methods for example there's now an FDA approved gene therapy treatment for
spinal muscular atrophy previously that disease was uniformly fatal to children by age two
but the seminal discoveries that led to that new gene therapy were made in our labs
the early versions of that treatment were manufactured here at the research institute
the first clinical trial that was done was hosted here at nationwide children's hospital
and we partnered with an industry partner to take those early accomplishments that we had done and
they got that treatment across the finish line and are now making it available to children around the
world that is the ultimate expression of what is exciting and possible now in pediatric research
and we are committed to doing that again and again one of the reasons for our early and continued
success in this endeavor is that we from from an early stage developed the capacity internally
to produce adeno-associated viruses that were suitable for human use in clinical trials and
this really is able to expedite our translation of gene therapy from the bench to the bedside with
this unmatched expertise and viral manufacturing for the trainee or postdoctoral researcher who's
thinking about coming to nationwide children's hospital this is a remarkable environment to do
training we have extraordinary resources here we have core resources that are critical to any
kind of scientific advancement scientific study that someone wants to do so we have
the tools and expertise and collaborators that are necessary and for the investigator this is a
special place in part because of the collegiality and collaboration of the rest of the faculty we
really have a culture that fosters integration and mutual support and that's really important
particularly for young investigators who are setting up a new laboratory it's a one-stop shop
because we have the state-of-the-art laboratories we have the vector manufacturing from research to
toxicology to clinical grade vector we have the support interacting with the regulatory
department and we have the support and engaging strategic partners we have a number of cores
including histology cores RNA sequencing cores bioinformatics cores immune monitoring cores
really almost any resource that you need is here and available to the investigators.