Gene Therapy Goals and Objectives
This program provides training for clinician-scientists in the field of genetic therapies for neurologic or neuromuscular diseases. It aims to ensure that by the end of the program, all trainees will:
- Be entirely conversant with modern molecular therapeutic approaches.
- Have completed IRB-certified training in Human Subjects Research.
- Have participated in performing at least one pre-clinical (laboratory-based) study of novel therapeutics with investigators within the Center for Gene Therapy.
- Have participated in the design, initiation, performance and/or interpretation of at least one clinical trial of genetic therapeutics within Nationwide Children's. These include trials either ongoing or in planning stages within the Center for Gene Therapy:
- AAV-mediated gene delivery
- Microdystrophin (gene replacement for DMD)
- U7snRNA (vectorized exon skipping for DMD)
- Alpha-sarcoglycan (LGMD)
- SGSH (MPSIIIB)
- IGHMBP2 (SMARD1/CMT2S)
- RNA-based therapeutics:
- 2’O-Methyl AON trial of exon 51 skipping in ambulant patients
- 2’O-Methyl AON trial of exon 51 skipping in non-ambulant patients
- Write and submit an NIH K08, K23 or equivalent foundation grant (e.g., American Academy of Neurology Clinical Research Training Grant), with a first submission by 14 months after joining the program
- Become an expert in the clinical, pathologic and molecular diagnosis of inherited neuromuscular diseases and demonstrate competencies to the satisfaction of Program Faculty and invited external reviewers.
- Demonstrate proficiency in basic laboratory techniques, including PCR, RT-PCR, Western Blot, primer design, immunofluorescent staining and sequence analysis (using common programs).
Scholarly Activity Expectations
All trainees are expected to pursue research projects they identify within the first two months in the program in collaboration with program faculty. They are expected to submit abstracts to two meetings per year. We anticipate that one of these will be the yearly meeting of the American Society of Gene and Cell Therapy. Each trainee is also expected to participate in the planning and/or performance of ongoing clinical trials of genetic therapies performed by the Center for Gene Therapy.
