Preliminary Conclusions obtained in Fanconi Anemia Patients Treated by Lentiviral-mediated Gene Therapy after more than 2 years of Follow-up
AAV9. LAMP-2B Reverses Metabolic and Physiologic Multiorgan Dysfunction in a Murine Model of Danon Disease
Systemic Delivery of AAV9.LAMP2B for the Treatment of Danon Disease: Toxicology Studies in Mice and Cynomolgus Monkeys
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
Poster: Gene Therapy for Leukocyte Adhesion Deficiency Type I (LAD-I) Preclinical Studies and Clinical Trial Design
Emerging Gene-Modified Cell Therapies
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