CRISPR/Cas9 AAV Production
The AAV-CRISPR system is particularly useful for editing disease-associated genes in the brain or central nervous system of mice due to the inability of most cationic nanocarriers to cross the blood-brain barrier, high transduction efficiency of AAV vectors in the brain, and non-dividing properties of neurons for long-term therapeutic effects. The AAV-CRISPR system has also been successfully used to restore the gene function in muscle-associated diseases such as Duchenne muscular dystrophy and diseases associated with the liver, heart, eye, and lung. This AAV-CRISPR-mediated gene editing in mice provided proof-of-principle studies for human disease modeling, gene therapy, or gene functional characterizations.
Created: 29 Oct 2024 09:10:43 AM
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