In a groundbreaking study, researchers utilized single-cell RNA sequencing to analyze the gene expression profiles of individual human embryonic stem cells as they underwent a 60-day differentiation protocol. This innovative approach not only enabled the mapping of transient states during the cells’ transformation into retinal pigment cells but also led to the optimization of the […]
Tag: gene
AAV Gene Therapy Vectors for Hemophilia
Recent research explores the theoretical risk of hepatocellular carcinoma (HCC) following adeno-associated virus (AAV) gene therapy in hemophilia patients. While data from clinical trials are reassuring, concerns remain about long-term oncogenic potential. A single case of HCC post AAV infusion in a patient with prior HCV infection raised questions, but further analysis suggested AAV integration […]
Gene Therapy Vectors for Hemophilia: Efficacy and Safety
Recent studies on gene therapy vectors for hemophilia have highlighted the varying effectiveness and risks associated with different AAV isotypes and gene constructs. Dosages ranging from 2 × 10^11 vector genome [vg]/kg to 6 × 10^13 vg/kg have been tested, showing significant reduction in bleeds for both hemophilia A and hemophilia B patients. Phase-1 trials […]
