Speaker articles

Take a look at what some of our Gene- and Cell-Based Therapies: CRISPR, Stem Cells, and Beyond speakers have published recently and chat with them about their work in San Francisco, USA

Keynote Speaker:

Alta Charo, UW Madison
Rejuvenating regenerative medicine regulation

Confirmed Speakers:

Daniel Anderson, Massachusetts Institute of Technology, USA
RNA Circularization Diminishes Immunogenicity and Can Extend Translation Duration In Vivo

Alessandra Biffi, Boston Children's Hospital, USA
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

Nathalie Cartier, INSERM, France
Clinical Gene Therapy for Neurodegenerative Diseases: Past, Present, and Future

Yvonne Chen, UCLA, USA
Rewiring T-cell responses to soluble factors with chimeric antigen receptors

Beverly Davidson, Perelman School of Medicine, University of Pennsylvania, USA
AAV-Mediated Progranulin Delivery to a Mouse Model of Progranulin Deficiency Causes T Cell-Mediated Toxicity

Viviana Gradinaru, Caltech, USA
Systemic AAV vectors for widespread and targeted gene delivery in rodents

Katherine High, Spark Therapeutics, USA
Hemophilia B gene therapy with a high-specific-activity factor IX variant

Anna Kajaste-Rudnitski, San Raffaele Telethon Institute for Gene Therapy, Ospedale San Raffaele, Italy
Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells

David Liu, Broad Institute, USA
Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors

Crystal MacKall, Stanford University, USA
Potent antitumor efficacy of anti-GD2 CAR T cells in H3-K27M+ diffuse midline gliomas letter

Eric Olson, UT Southwestern Medical Center, USA
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

Malin Parmar, Lund University, Sweden
Target-specific forebrain projections and appropriate synaptic inputs of hESC-derived dopamine neurons grafted to the midbrain of parkinsonian rats

Matthew Porteus, Stanford University, USA
Highly Efficient and Marker-free Genome Editing of Human Pluripotent Stem Cells by CRISPR-Cas9 RNP and AAV6 Donor-Mediated Homologous Recombination

Waseem Qasim, University College London, UK
Long Terminal Repeat CRISPR-CAR-Coupled “Universal” T Cells Mediate Potent Anti-leukemic Effects

Sonja Schrepfer, UCSF, USA
Hypoimmunogenic derivatives of induced pluripotent stem cells evade immune rejection in fully immunocompetent allogeneic recipients

Luk Vandenberghe, Harvard Medical School, USA
The Assembly-Activating Protein Promotes Stability and Interactions between AAV's Viral Proteins to Nucleate Capsid Assembly


Jennifer Doudna, UC Berkeley, UCSF Gladstone Institutes, USA
A Functional Mini-Integrase in a Two-Protein-type V-C CRISPR System

Luigi Naldini, San Raffaele Telethon Institute for Gene Therapy, Italy
Phagocytosis-shielded lentiviral vectors improve liver gene therapy in nonhuman primates


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