Donald R. Wassenberg II

Eric Olson, Ph.D.
Professor and Founding Chair of the Department of Molecular Biology
UT Southwestern Medical Center
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Skeletal and Cardiac Muscle Development and Genetic Disease Therapy

Olson directs the Hamon Center for Regenerative Science and Medicine and the Wellstone Center for Muscular Dystrophy Research. His lab discovered key regulators of muscle development (myogenin, MEF2, Myomaker, Myomixer, Hand factors, myocardin) and stress-response pathways. His latest work uses CRISPR to correct Duchenne muscular dystrophy. He’s published 600+ articles (200K+ citations), is a member of NAS, NAM, AAAS, and co-founded multiple biotech firms.

Diana Bautista, Ph.D.
Professor & Head, Division of Cell Biology, Developmental Biology & Physiology
UC Berkeley
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Peripheral Neuroimmune Signaling in Eczema

Dr. Bautista studies neuroimmune interactions in chronic pain, itch, and airway inflammation, focusing on TRPA1 and TRPM8 sensory ion channels. She’s an HHMI Investigator, Pew Scholar, NIH Director’s Transformative Research Awardee, and a leader in DEI mentorship.

Nancy Bonini, Ph.D.
Florence R.C. Murray Professor, Biology
University of Pennsylvania
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Drosophila as a Model for Human Neurodegenerative Disease

Bonini uses Drosophila to uncover mechanisms of neurodegenerative diseases. Her pioneering work earned her NAS, NAM, AAAS membership, Packard and Merck awards, and HHMI Investigator status.

Paul Turner, Ph.D.
Rachel Carson Professor of Ecology & Evolutionary Biology
Yale University
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Phage Therapy for CF Patients

Turner researches virus evolution and phage therapy for antibiotic-resistant infections in cystic fibrosis. He’s active in STEM outreach, served on NSF and society boards, and is a NAS fellow.

Madelon Maurice, Ph.D.
Professor of Molecular Cell Biology
UMC Utrecht, The Netherlands
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Mechanisms of Wnt Pathway Tumor Suppressor Mutations

Maurice uncovered how Wnt signaling misregulation drives cancer. Her work earned ERC and NWO VICI grants and Oncode Institute membership.

Leslie Leinwand, Ph.D.
Distinguished Professor of MCDB & CSO, BioFrontiers
Colorado Boulder
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Diseases Caused by Myosin Mutations

Leinwand studies cardiomyopathies and gender/diet effects on heart disease. She’s Fellow of AAAS, NIH MERIT awardee, AHA Established Investigator, NAS and National Academy for Inventors member.

Paul Negulescu, Ph.D.
SVP, Research & SD Site Head
Vertex Pharmaceuticals
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Can We Fix the Broken Protein in CF?

Negulescu led development of Kalydeco (2012) and Orkambi (2015). Under his leadership, Vertex’s SD site generated multiple approved therapies for cystic fibrosis.

Joseph C. Wu, M.D., Ph.D.
Director, Stanford Cardiovascular Institute
Stanford University
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Cardiac iPSCs for Precision Medicine

Wu’s lab uses iPSCs and molecular imaging to model cardiovascular disease and develop gene/cell therapies. Awards include NIH New Innovator and Presidential Early Career Award.

Forest Rohwer, Ph.D.
Department of Biology, SDSU
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Personalized Medicine Platform for CF

Rohwer pioneered “viromics” to profile viral diversity. Applied to CF lungs, his work reveals overlooked microbes and informs personalized treatment. Published >150 articles; 2008 ISME Young Investigator; author of two books.

John M. Carethers, M.D.
John G. Searle Professor & Chair, Internal Medicine
University of Michigan
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DNA Mismatch Repair and Hereditary Colorectal Cancer

Carethers researches tumor genetics, DNA repair, and colorectal cancer disparities. He’s IOM member, Senior Associate Editor of “Gastroenterology,” and NIH-funded investigator with 100+ publications.

Mario R. Capecchi, Ph.D.
Dept of Human Genetics
University of Utah
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Gene Targeting into the 21st Century

Capecchi developed ES cell gene targeting, revolutionizing mouse models of human disease. Nobel Laureate; research in neural development, gene therapy, and genomic rearrangements.

Nigel G. Laing, Ph.D.
Head of Neuromuscular Diseases
Centre for Medical Research, UWA
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Next-Gen DNA Sequencing: Gene Discovery & Diagnostics

Laing identified 20+ neuromuscular disease genes. His lab advanced molecular neurogenetics diagnostics, including nemaline myopathy and distal myopathy.

K. David Becker, Ph.D.
Sr Director, Lab Ops
Helix, San Diego
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Translational Science, Clinical Medicine & Personal Genomics

Becker led clinical lab and FDA affairs at Pathway Genomics. His research spans muscle disease models and Alzheimer’s genomics.

Albert La Spada, M.D., Ph.D.
Head, Genetics
UCSD School of Medicine
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Dynamic Mutation & Neurodegenerative Repeat Expansion Disorders

La Spada’s lab studies molecular pathology of SCAs and Kennedy’s disease using model organisms to develop therapies. Funded by NIH, HHMI, and MDA.

Atul Butte, M.D., Ph.D.
Professor, UCSF School of Medicine
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Exploring Genomic Medicine Using Translational Bioinformatics

Butte’s lab develops computational approaches to integrate clinical EHR data with gene expression. He founded Genstruct, and has published extensively in Nature-group journals.

Garry P. Nolan, Ph.D.
Professor of Microbiology & Immunology
Stanford University
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Phospho-Biosignatures and Disease

Nolan’s group pioneered single-cell proteomics and engineered viral cloning systems. Recipient of Stohlman Scholar Award and Burroughs Wellcome Award; founded multiple biotech companies.

Jeffrey Robbins, Ph.D.
Exec Co-Director, The Heart Institute
Cincinnati Children’s Hospital
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Progenitor Cell Populations: the Birth of a Cardiac Syndrome

Robbins studies genetic, cell biological, and protein-level mechanisms of cardiac disease. AHA MERIT awardee and Presidential Award for Research from ISHR; 160+ publications.

Jeffrey S. Chamberlain, Ph.D.
Director, Wellstone MD Cooperative Research Center
Univ of Washington
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Development of Gene Therapy for the Muscular Dystrophies

Chamberlain’s lab develops gene/cell therapies for muscular dystrophy. NIH MERIT award recipient; editorial boards, and >160 publications in Nature/Science journals.

Andrew D. McCulloch, Ph.D.
Distinguished Professor of Bioengineering
UC San Diego
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Cardiac Systems Biology

McCulloch uses experimental/computational models to study cardiac muscle structure–function relationships and arrhythmia. NSF Young Investigator and AIMBE Fellow; co-founded Insilicomed.

Christine E. Seidman, M.D.
Professor & Director, Cardiovascular Genetics Center
Brigham & Women’s Hospital/Harvard Medical School
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Genetic Causes of Cardiac Hypertrophy

Seidman’s research on cardiomyopathies has defined the genetic basis for human heart disease. Bristol-Myers Squibb Awardee; 200+ publications; NAS and IOM member.

Stuart H. Orkin, M.D.
Chair, Pediatric Oncology & Professor of Pediatrics
Dana-Farber/Harvard Medical School
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Genetic Control of Blood Cell Development

Orkin defined hematopoietic transcriptional regulators. HHMI Investigator; NAS and IOM member; editorial boards; 300+ primary research articles.

Jean Y. J. Wang, Ph.D.
Professor of Medicine & Associate Director, Basic Research
Moores UCSD Cancer Center
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Exploiting an Intrinsic Vulnerability of Uncontrolled Proliferation in Cancer

Wang’s work on proto-oncogenes and signaling networks under genotoxic stress revealed new cancer gene interactions and therapeutic targets. Jane Coffin Childs Fellow; Searle Scholar; 100+ publications.

Phyllis I. Gardner, M.D.
Professor of Medicine & Clinical Pharmacology
Stanford School of Medicine
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The Evolving Impact of the Genomic Revolution on Molecular Diagnostics and Therapies

Gardner’s research on ion channel signaling includes CF chloride channels. Founder of Genomics Collaborative, CambriaTech, Xeragen. Board member of biotech firms; Harvard Med graduate.

Theodore Friedmann, M.D.
Prof of Pediatrics & Chair of Biomedical Ethics
UC San Diego School of Medicine
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Progress Toward Human Gene Therapy: Technology, Policy and Ethics

Friedmann pioneered human gene therapy protocols. Director of UCSD Human Gene Therapy Program; member of NIH ethics committees; Muriel Jeannette Whitehill Chair.

Richard Kolodner, Ph.D.
Prof of Medicine & Head, Cancer Genetics
Ludwig Institute/UC San Diego
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Mutator Genes, Genome Instability and Cancer Susceptibility

Kolodner elucidated DNA repair and recombination mechanisms in yeast and mammals, defining their roles in cancer. Former Harvard faculty; associate editor of Cell; NAS member.

Neal D. Epstein, M.D., Ph.D.
Chief, Molecular Physiology Section
NIH NHLBI, Bethesda

Of Flies and Men: Lessons from Hypertrophic Cardiomyopathy

Epstein studies molecular physiology of cardiomyopathies, translating insights from model organisms to human disease. Recipient of PHS Outstanding Service Medal.

Owen N. Witte, M.D.
Director, Broad Center for Regenerative Medicine & Stem Cell Research
UCLA
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Lymphocyte Signal Transduction through Bruton’s Tyrosine Kinase

Witte discovered the role of Btk in immune development. HHMI Investigator; National Academy of Sciences member.

Philip C. Hanawalt, Ph.D.
Professor of Biology & Dermatology
Stanford University

Transcription-Coupled DNA Repair & Human Disease

Hanawalt discovered transcription-coupled repair pathways, explaining DNA repair defects in xeroderma pigmentosum. NAS member; editorial boards of Science and six other journals.

David S. Bredt, M.D., Ph.D.
Global Head, Neuroscience Discovery
Johnson & Johnson, San Diego

Nitric Oxide Synthase and Duchenne Muscular Dystrophy

Bredt characterized nNOS roles in neuromuscular disease and Alzheimer’s research. Led neuroscience at Eli Lilly and J&J; AAM and AAAS fellow.

Roger W. Wiseman, Ph.D.
Lead, Molecular Carcinogenesis Group
NIEHS

Identification of BRCA1 – Promises and Paradoxes

Wiseman studies BRCA1 functions in breast/ovarian cancer susceptibility. Presented at Gordon Research Conferences and Human Genetics Congress; funded by NSF and NIH.

William B. Guggino, Ph.D.
Director, CF Research Development Program & Chair, Physiology
Johns Hopkins University
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The Molecular Defect in CF: Can We Correct It?

Guggino characterized CFTR chloride channel defects and pioneered gene/drug therapy approaches to restore function.

Inder M. Verma, Ph.D.
ACS Professor of Molecular Biology
Salk Institute
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Prospects in Human Gene Therapy

Verma developed retroviral gene-transfer vectors and defined oncogene functions, paving the way for gene therapy trials. Edited PNAS; chaired biotech advisory boards.

Henry F. Epstein, M.D.
Professor, Neuroscience & Cell Biology
UT Galveston

Genetic Advances in Human Muscle Disease

Epstein’s C. elegans work uncovered myofibril assembly and myotonic dystrophy mechanisms; co-director of Baylor Neuromuscular Disease Center.

Savio Woo, Ph.D.
Professor of Medicine & Oncology
Mt. Sinai

Molecular Genetics of PKU: From Anthropology to Gene Therapy

Woo founded Baylor’s Center for Gene Therapy; pioneered gene therapy approaches for PKU; HHMI Investigator.

Charles D. Laird, Ph.D.
Professor Emeritus of Biology
University of Washington

Applying Drosophila Chromosome Principles to Fragile-X Syndrome

Laird transitioned from insect chromosome structure to defining human FMR1 molecular pathology in Fragile-X syndrome. Supported by NSF, NIH, Kennedy Foundation.

Francis S. Collins, M.D., Ph.D.
Director, NIH
Profile

Progress in the Search for the Cystic Fibrosis Gene

Collins led identification of CFTR. HHMI Investigator; Chief of Medical Genetics at Michigan; studies neurofibromatosis and Huntington’s disease.