Gainesville, FL—Much work is being done with Adeno-associated virus (AAV) vectors for application in retinal gene therapy, with modifications being made to the capsid and genome of the vector to generate novel variants with unique transduction profiles, according to Shannon Boye, PhD.
“The AAV vector toolkit is expanding rapidly,” said Dr. Boye, assistant professor, University of Florida, Gainesville.
Sequence differences in the large variable regions of the AAV capsid account for functional differences between serotypes in receptor biology and immunologic profiles, among others.
Rational design and directed evolution are two methods used to modify the variable regions, she explained. The former involves using knowledge of vector biology to make structure-informed mutations to the capsid sequence to generate novel variants, e.g., the tyrosine-phenylalanine story led by Arun Srivastava, PhD, at the University of Florida.