Previous efforts to circumvent the problem of the size limitation imposed by the AAV vector have focused mainly on altering the candidate genes into ‘mini-expression cassettes’ suitable for ...

AI-assisted AAV capsid engineering has transformative potential in terms of accelerating and optimizing the development of gene therapies.

"Protein design is a hard problem because the mapping from DNA sequence to protein structure and function ... for the viral ...

For central nervous system (CNS) applications, attempts are being made to deliver AAV gene therapy directly to the brain parenchyma or the cerebrospinal fluid (CSF) space by intra-cisterna magna ...