ASGCT Presentations & Posters

A Phase 1/2 Study of Lentiviral-mediated Ex vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency I (LAD I): Initial Results from the First Treated Patient (click to download)

Updated Results of a European Gene Therapy Trial in Fanconi Anemia Patients, Subtype A (click to download)

Poster: Lentiviral Vector-mediated Gene Complementation for the Diagnosis of LAD-I (click to download)

Preliminary Conclusions obtained in Fanconi Anemia Patients Treated by Lentiviral-mediated Gene Therapy after more than 2 years of Follow-up (click to download)

AAV9. LAMP-2B Reverses Metabolic and Physiologic Multiorgan Dysfunction in a Murine Model of Danon Disease (click to download)

Systemic Delivery of AAV9.LAMP2B for the Treatment of Danon Disease: Toxicology Studies in Mice and Cynomolgus Monkeys (click to download)

Poster: Finding the Minimum Vector Copies Per Cell Needed to Reach Phenotypic Correction in a Mouse Model of Erythrocyte Pyruvate Kinase Deficiency using a Clinically Applicable Lentiviral Vector (click to download)

Poster: Finding the Minimum Vector Copies Per Cell Needed to Reach Phenotypic Correction in a Mouse Model of Erythrocyte Pyruvate Kinase Deficiency using a Clinically Applicable Lentiviral Vector (click to download)

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