Tyrosinemia, also referred to in the literature as tyrosinosis or tyrosyluria, is a disease characterized by elevated levels of sermn tyrosine and by the presence of exce [...]

Adeno-associated virus (AAV) is widely used in gene therapy but has limited integration, reducing long-term transgene expression. We engineered an AAV-rDNA vector to enha [...]

Orthotopic liver transplantation effectively treats pediatric metabolic diseases caused by hepatocyte-specific enzyme deficiencies, but donor scarcity, high cost, and lif [...]