Neuromuscular Genetic Therapeutics Fellowship Goals and Objectives

Our Fellowship provides training for clinician scientists in the field of genetic therapies for neuromuscular disease.  

At the end of training, the specific goals are that all trainees will:

  • Be entirely conversant with modern molecular therapeutic approaches.

  • Have completed IRB-certified training in Human Subjects Research. 

  • Have participated  in the performance of 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 Hospital.  These include trials either ongoing or in planning stages within the Center for Gene Therapy:

    • AAV-mediated gene delivery

      • Follistatin (funded; IND approved)

      • Microdystrophin (funded; pre-IND work underway)

      • Alpha-sarcoglycan (clinical study funded; pre-IND work underway)

      • NAGLU (MPSIIIB) (pre-clinical IND enabling studies funded and underway)

      • GALGT2 (pre-clinical IND enabling studies funded and underway)

    • Exon skipping:

      • 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 (American Academy of Neurology Clinical Research Training Grant).

  • Become an expert in the clinical, pathologic, and molecular diagnosis of inherited neuromuscular diseases.  Trainees will 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 will be expected to pursue research projects, to be identified within the first two months in the program, in collaboration with program faculty.  They will be 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 will be expected to participate in the planning and/or performance of ongoing clinical trials of genetic therapies being performed by the Center for Gene Therapy.

Our goal for all trainees will be to have them write a K08 or K23-level award (or their equivalent, in some cases, from private foundations), with a first submission by 14 months after joining the program.