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Research

Superb care of the fetal and pediatric patient and family requires not only productive clinical interaction of disparate clinical disciplines, but successful collaboration of diverse biological and physical science groups in the research laboratory. In our paradigm of surgical care, the problems of our small patients also become the motivation for investigators in the basic sciences to assist in the understanding of disease mechanism and develop new therapies to cure, ameliorate, or prevent previously untreatable birth defects.

In Search of Innovative Solutions

An underlying premise of our research is the assumption that the current relatively primitive, albeit successful, surgical approaches to the correction of structural birth defects will be complemented and in many cases supplanted by molecular, cell, or organ transplantation in the near future. The focus of our research is in areas that have great promise of radically changing the way we will diagnose and treat human fetuses, newborns, and children with birth defects over the next decade. Our research program builds on existing strengths already present with our lab and others at UCSF, develops links to private biotechnology firms, and emphasizes translation to innovative human therapies. 

Invasive fetal and pediatric surgery for severe anatomic defects may have been the starting point for our program, but it was only the beginning. Our clinical and basic science research shaped and continues to pave for the future the use of minimally or non-invasive techniques such as:

  • percutaneous and endoscopic fetal and pediatric surgery
  • radiofrequency ablation of some fetal tumors
  • laser ablation of communicating vessels in twin-twin transfusion sydrome
  • stem cell transplantation for diseases of the blood (thalassemia, sickle cell anemia)
  • brain (neurodegenerative diseases)
  • liver (urea cycle and other inborn errors)
  • tissue engineering for deficient organs (coverage of myelomeningocele, bone grafts for craniofacial disorders)
  • local molecular therapies (growth and anti-angiogenisis factors for cleft lip and palate, CCAM, SCT)
  • gene therapy for single gene disorders (hemophilia).

We aim to be at the forefront of this transition just as we led the current efforts of aggressive intervention for surgically correctable birth defects.

New Surgical Tools & Techniques

While many of the innovative fetal intervention techniques being applied around the world were developed in our own Pediatric Surgery & Fetal Treatment Research Laboratory, we continue our constant technical innovation in a variety of animal models as well as use of experimental tools to push fetal intervention in new directions that go well beyond traditional surgical techniques:  microsurgical fetoscopy, three-dimensional image-guided sampling and manipulation using sonography and MRI, videoendoscopic techniques using virtual reality and robotic manipulation, placental vessel catheterization for stem cell transplantation and gene therapy, and microsurgical techniques for gene, cell, and organ transplantation. 

For an overview of our current clinical trials, visit Pediatric Surgery Clinical Trials.

More information on our research can be found on the Pedatric Surgery & Fetal Treatmnent Lab website or the Fetal Treatment Center Research page.

 

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