Enhanced AAV production via rational design of a novel pHelper vector integrated with HSV-1 helper genes.

ABSTRACT: Adeno-associated virus (AAV) vectors are widely used in gene therapy owing to their safety, stability, and broad tissue tropism. However, current plasmid-based AAV manufacturing platforms suffer from low yield and high manufacturing cost, limiting their scalability for clinical and commercial applications. Rational engineering of pHelper vector offers an effective strategy to enhance AAV production. In this study, we engineered a novel helper vector (UL12-ICP22-miniHelper) by integrating UL12 and ICP22 genes from herpes simplex virus type 1 (HSV-1) into a size-reduced pHelper backbone (mini-pHelper) with partial deletion of E2a and E4 regions. In a triple-plasmid transfection system, UL12-ICP22-miniHelper increased AAV5 vector yield from 1.35 × 10¹¹ t

SUBMITTER: Ren H 

PROVIDER: S-EPMC12830312 | biostudies-literature | 2026 Jun

REPOSITORIES: biostudies-literature