Projects

Current Research Projects

Natural History Study of Cockayne Syndrome

Funded by: National Initiative for Cockayne Syndrome (NICS)

Start Date: September 1, 2022

End Date: September 1, 2023

Foundations for a Phase 1 Clinical Trial of Cell-Based Therapy for Duchenne Muscular Dystrophy

Funded by: National Institutes of Health - National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIH/NIAMS)

Start Date: July 15, 2022

End Date: June 30, 2024

GRASP-LGMD: Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy Subtypes 2E, 2D, 2F, 2C

Funded by: Virginia Commonwealth University

Start Date: October 30, 2021

End Date: October 29, 2023

Local site for NCT03981289

Optimized Production and Validation of Rationally Designed AAV Vectors for Cockayne Syndrome Gene Therapy

Role: Co-Investigator (PIs: Christina Pacak & Georgiy Aslanidi)

Funded by: United Stated Food & Drug Administration (FDA)

Start Date: September 1, 2021

End Date: August 31, 2024

Read more >

Completed Projects

Surveillance of Muscular Dystrophy in the Southeastern United States

Start Date: January 1, 2021

End Date: August 31, 2022