Viral gene therapeutic strategies to obtain abluminal protein secretion from brain endothelial cells denoting the blood-brain barrier
Muscle-targeted Klotho Gene Therapy Ameliorates ALS Hallmarks by Addressing Multiple Disease Mechanisms in SOD1G93A Mice
A new autophagy-related nomogram and mechanism in multiple myeloma
Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia
Prevalence of antibodies against adeno-associated viruses (AAVs) in Göttingen Minipigs: Implications for Gene Therapy and Xenotransplantation
Liver fibrosis negatively impacts in vivo gene transfer to hepatocytes
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice.
Engineering Tripartite Gene Editing Machinery for Highly Efficient Non-Viral Targeted Genome Integration
Precise genetic control of ATOH1 enhances maturation of regenerated hair cells in the mature mouse utricle
Lipopolymer Mediated siRNA Delivery Targeting Aberrant Oncogenes for Effective Therapy of Myeloid Leukemia