Marek Napierala

Marek Napierala, Ph.D. is a Professor in the Department of Neurology at The University of Texas Southwestern Medical Center, part of the Peter O'Donnell Jr. Brain Institute. He joined UT Southwestern in 2022 after serving as an Associate Professor at the University of Alabama at Birmingham. His research focuses on molecular mechanisms underlying Friedreich's Ataxia (FRDA), a neurodegenerative disorder caused by expanded GAA repeat sequences in the FXN gene. Dr. Napierala's work integrates cellular and mouse models, induced pluripotent stem cells (iPSCs), and genome editing to study FRDA pathogenesis and therapeutic strategies.

Education:

• M.S. in Biotechnology, Adam Mickiewicz University, Poland (1990s)
• Ph.D. in Biochemistry, Institute of Bioorganic Chemistry, Polish Academy of Sciences (1999)

Research Interests:

• Mechanisms of GAA repeat expansion and transcriptional silencing in FRDA
• Development of gene therapy and antisense oligonucleotide approaches
• CRISPR-based genome editing for FRDA correction
• Identification of biomarkers and disease progression indicators

Key Contributions:

• Established the Friedreich’s Ataxia Cell Line Repository, the world's largest fibroblast and iPSC bank from FRDA patients.
• Advocacy and collaboration with FRDA patient communities, including annual patient meetings and participation in rideATAXIA events.
• Member of the Scientific Advisory Board of the Friedreich’s Ataxia Research Alliance (FARA).

Labs & Teams: The Napierala Lab focuses on multidisciplinary approaches to FRDA, including iPSC differentiation into neurons and cardiomyocytes, CRISPR-based editing, and translational drug discovery. Collaborations include the Peter O'Donnell Jr. Brain Institute and global FRDA research networks.