Scientific Session

12:00pm Mitochondria and Metabolism Platform – Part 1
Session Moderator:  Brett Kaufman, PhD, University of Pittsburgh, Pittsburgh, PA

  • Mitochondrial Systems Biochemistry
    Dave Pagliarini, PhD, Washington University School of Medicine, St. Louis, MO
  • Targeting Cancer Asparagine Dependence
    Heather Christofk, PhD, University of California Los Angeles, CA
  • TCA Cycle Fuels and Fluxes
    Josh Rabinowitz, MD, PhD, Princeton University, Princeton, NJ

           Three 30-minute presentations with Live Q&A


1:30pm Abstract Presentations with Live Q&A

  • Synthetic Messenger RNA Rescues Very Long-Chain Acyl-CoA Dehydrogenase Deficiency in Fibroblasts from Patients and a Murine Model, Xuejun Zhao
  • Maladaptive Nutrient Signaling Sustains the m.3243A>G mtDNA Mutation, Chih-Yao Chung
  • The gnomAD Database of Mitochondrial DNA Variation from 56,434 Individuals, Sarah Calvo

12:00pm Mitochondria and Metabolism Platform – Part 2
Session Moderator:  Carla Koehler, PhD, University of California Los Angeles, Los Angeles, CA

  • Mitochondria in Alzheimer Disease: It's Not What You Think
    Eric Schon, PhD, Columbia University, New York, NY
  • Mitochondria, Metabolism and Cellular Decisions
    Jared Rutter, PhD, University of Utah, Salt Lake City, UT
  • Enlightening Mechanisms of Mitochondrial Encephalopathies
    Anu Suomalainen-Wartiovaara, MD, PhD, University of Helsinki, Finland

           Three 30-minute presentations with Live Q&A


1:30pm Abstract Presentations with Live Q&A

  • A Cardiolipin-Binding Peptide Improves Cellular Bioenergetics in Mitochondrial Trifunctional Protein (TFP)-Deficient Mice and Patient Fibroblasts, Meicheng Wang
  • Mitochondrial Protein Import Clogging as a Mechanism of Disease, Liam Coyne
  • SLC25A46 Organizes a Platform for Mitochondrial Membrane Dynamics and Lipid Remodeling, Jana Schuettpelz

12:00pm Gene Therapy/Gene Editing Platform
Session Moderator:  Giovanni Manfredi, MD, PhD, Weill Cornell Medicine, New York, NY

  • Progress on Gene Therapy for mtDNA Diseases
    Carlos Moraes, PhD, University of Miami, Miami, FL
  • Gene Therapy for SURF1-related Leigh Syndrome
    Steven Gray, PhD, University of Texas Southwestern Medical Center, Dallas, TX
  • Topic - Title TBD
    Mickael Decressac, PhD, Grenoble Institute of Neuroscience (GIN), France

           Three 30-minute presentations with Live Q&A


1:30pm Abstract Presentations with Live Q&A

  • Modulation of the Nuclear Epigenome as a New Strategy for Mitochondrial DNA Heteroplasmy Shift, Lia Mayorga
  • Assessment of Lenadogene Nolparvovec (rAAV2/2-ND4) Gene Therapy Efficacy in Leber Hereditary Optic Neuropathy Using an External Control Group, Mark Moster
  • Gene Replacement Therapy for SURF1-Related Leigh Syndrome Using AAV9, Qinglan Ling

12:00pm Mitochondria in Stress Platform
Session Moderator:  Robert K. Naviaux, MD, PhD, University of California San Diego, La Jolla, CA

  • Mitochondrial Behavior
    Jodi Nunnari, PhD, University of California Davis, Davis, CA
  • Hypoxia as a Therapy for Mitochondrial Disease
    Vamsi Mootha, MD, Harvard Medical School, Boston, MA
  • Mitochondrial Control of Innate Immunity and Inflammation
    Phillip West, PhD, Texas A&M University College of Medicine, Bryan, TX

           Three 30-minute presentations with Live Q&A


1:30pm Abstract Presentations with Live Q&A

  • Characterization of Two Variants in the Mitochondrial Topoisomerase Gene TOP1MT Reveal a Potential Involvement in Human Pathology, Iman Al Khatib
  • Identification of Novel Compounds to Treat Rare Mitochondrial Diseases, Arwen Gao
  • Vaccine Hesitancy toward the COVID19 Vaccine and Attitudes toward Ring Vaccination by Caregivers of Children with Mitochondrial Disease, Eliza Gordon-Lipkin