801. Gene Therapies: Poster II

Transient ATM inhibition enhances knock in efficiency and long term engraftment of genome edited hematopoietic stem cells

First in human prime edited autologous hematopoietic stem cell therapy for the treatment of p47phox CGD Initial results of PRIME 0101

Low dose AAV8 FVIII gene therapy on Chinese severe Hemophilia A patients with sustainable efficacy and safety

Transduction efficiency and vector biodistribution following prenatal vs neonatal AAV5 gene therapy for Hemophilia A in a sheep model

Adeno associated viral integration profiles in human liver biopsies following Hemophilia A gene therapy

CRISPR Cas12a gene editing of the HBG1 2 promoters leads to sustained normalization of total hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease Updated Results from the RUBY trial

Lentiviral hematopoietic stem and progenitor cell gene therapy for Wiskott Aldrich Syndrome Early access experience in Italian patients based on 648 1996 law

Activated factor IX Ρadua delivered via adeno associated virus enhances therapeutic efficacy in hemophilia B

Decoding Transgene Silencing from Stably Maintained AAV Genomes in Human Liver Epigenetic Insights to Guide Next Generation Vector Design