Jan 1, 2024 · Among the AAV vectors, AAV serotype 8 (AAV8) has attracted much attention for its efficient and stable gene transfection into specific tissues. Currently, recombinant AAV8 has been widely used in gene therapy research on a variety of diseases, including genetic diseases, cancers, autoimmune diseases, and viral diseases.

AAV serotype 8 (AAV8) shows a significantly greater liver transduction efficiency than those of other serotypes, which has resulted in efforts to develop this virus as a gene therapy vector for hemophilia A and familial hypercholesterolemia.

Eleven serotypes of AAV have thus far been identified, with the best characterized and most commonly used being AAV2. These serotypes differ in their tropism, or the types of cells they infect, making AAV a very useful system for preferentially transducing specific cell types.

AAV serotype 8 (AAV8) shows a significantly greater liver transduction efficiency than those of other serotypes, which has resulted in efforts to develop this virus as a gene therapy vector for hemophilia A and familial hypercholesterolemia.

Mar 14, 2024 · Fonck and colleagues report that AAV8-mediated systemic administration of FXN transgene significantly improved survival, body weight, heart morphology, cardiac function, cardiac injury biomarkers, and expression of mitochondrial electron transport chain complexes of Friedreich’s ataxia without toxicity observed in the heart and liver of a FXN ...

AAV serotype 8 (AAV8) in particular has been demonstrated to be one of the most effective vectors in some structures of the CNS, producing the highest rate of transgene transduction in the striatum compared with other serotypes, in the absence of neurotoxicity (Aschauer et al., 2013).

Feb 27, 2005 · We show that AAV8 is the most efficient vector for crossing the blood vessel barrier to attain systemic gene transfer in both skeletal and cardiac muscles of mice and hamsters.

We have recently isolated and characterized an AAV8-specific monoclonal antibody, ADK8 (67), which efficiently neutralizes AAV8 gene transduction in vitro (Fig. 6A) and also in vivo in mice (20a).

Jan 12, 2024 · Research has indicated that AAV8 and AAV9 exhibit a notable affinity for liver cells, with AAV8 demonstrating the highest degree of hepatophilia. rAAV8 can transfect hepatocytes in primates, canines, and rodents with high efficiency and stability through intraperitoneal injection, portal vein, or peripheral vein.

Mar 10, 2022 · Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical physiological functions and involvement in a wide range of genetic diseases.