The goal of this T32 Training Program proposal is to facilitate the development of a new generation of medical and surgical fetal specialists that will be well trained to carry out innovative and scientifically rigorous laboratory and clinical research directed toward novel and effective strategies for fetal treatment. The need for such a program is highlighted by the present lack of specific subspecialty training and preparation of investigators for treatment of the fetus as a patient. At the same time, the perception of the fetus as a patient is gaining momentum, and the number of claimed successful interventions applicable to the fetus has increased rapidly. There is thus a compelling need for well trained physician scientists in this field for developmental research and analysis of efficacy and outcomes. The Program will support Trainees for up to 3 years of clinical or laboratory research training at the postdoctoral fellowship level. The Training faculty includes 16 Faculty Mentors from The Children's Hospital and the University of Pennsylvania School of Medicine and 1 from Cornell University, who have outstanding credentials and a focused interest in laboratory or clinical research directed toward fetal treatment. These mentors will supervise Trainees in basic laboratory research and/or patient-oriented research projects related to fetal treatment. Research opportunities will include four major areas of basic research 1) Stem cell therapy in the fetus; 2) Fetal gene therapy; 3) Fetal wound healing; and 4) Fetal anatomic malformations and fetal lung growth. Patient oriented research opportunities will be derived from the CHOP Center for Fetal Diagnosis and Treatment and will include opportunities derived from two existing NICHD sponsored multicenter, randomized, clinical trials examining fetal treatment of myelomeningocele and Twin-Twin Transfusion Syndrome at CHOP as well as participation in the design and implementation of new studies of various aspects of fetal therapy. The Program includes training in all aspects of research, including biostatistics and bioethics, with a specific program in ethics in the context of fetal therapy, molecular biology, and related. The Program is strongly supported by access to a superb range of institutional resources, including the CHOP Center for Fetal Diagnosis and Therapy, the CHOP GCRC and the U. Penn Graduate School and IHGT. This request is made for 2 Trainee slots in year 1, 3 Trainee slots in year 2, and 4 Trainee slots each year thereafter.

Agency
National Institute of Health (NIH)
Institute
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Type
Institutional National Research Service Award (T32)
Project #
5T32HD046402-02
Application #
6859362
Study Section
Pediatrics Subcommittee (CHHD)
Program Officer
Reddy, Uma M
Project Start
2004-05-01
Project End
2009-04-30
Budget Start
2005-05-01
Budget End
2006-04-30
Support Year
2
Fiscal Year
2005
Total Cost
$177,399
Indirect Cost
Name
Children's Hospital of Philadelphia
Department
Type
DUNS #
073757627
City
Philadelphia
State
PA
Country
United States
Zip Code
19104
Roybal, J L; Endo, M; Radu, A et al. (2012) Early gestational gene transfer with targeted ATP7B expression in the liver improves phenotype in a murine model of Wilson's disease. Gene Ther 19:1085-94
Roybal, J L; Endo, M; Radu, A et al. (2011) Early gestational gene transfer of IL-10 by systemic administration of lentiviral vector can prevent arthritis in a murine model. Gene Ther 18:719-26
Peranteau, William H; Heaton, Todd E; Gu, Yu-Chen et al. (2009) Haploidentical in utero hematopoietic cell transplantation improves phenotype and can induce tolerance for postnatal same-donor transplants in the canine leukocyte adhesion deficiency model. Biol Blood Marrow Transplant 15:293-305
Badillo, Andrea T; Beggs, Kirstin J; Javazon, Elisabeth H et al. (2007) Murine bone marrow stromal progenitor cells elicit an in vivo cellular and humoral alloimmune response. Biol Blood Marrow Transplant 13:412-22
Javazon, Elisabeth H; Keswani, Sundeep G; Badillo, Andrea T et al. (2007) Enhanced epithelial gap closure and increased angiogenesis in wounds of diabetic mice treated with adult murine bone marrow stromal progenitor cells. Wound Repair Regen 15:350-9
Peranteau, William H; Endo, Masayuki; Adibe, Obinna O et al. (2007) Evidence for an immune barrier after in utero hematopoietic-cell transplantation. Blood 109:1331-3
Peranteau, William H; Endo, Masayuki; Adibe, Obinna O et al. (2006) CD26 inhibition enhances allogeneic donor-cell homing and engraftment after in utero hematopoietic-cell transplantation. Blood 108:4268-74