Identification of Adeno-Associate Virus (AAV) Serotype for Endometriosis Therapy and Effect of AAV-Mediated RNAi Delivery on Gene Expression and Cell Proliferation in In Vitro Endometrial Cell Culture.

ABSTRACT: Endometriosis is a chronic estrogen-dependent condition with limited treatment options, often requiring surgery and long-term hormonal therapy that may impair ovarian function. Despite advancements in gene therapy for other diseases, its application in endometriosis remains largely unexplored. This study aimed to evaluate the potential of adeno-associated virus (AAV) vectors for targeted gene therapy in endometriosis. We screened multiple AAV serotypes for infectivity in primary human ectopic and eutopic endometrial cells as well as normal ovarian stromal cells. AAV serotype 3 (AAV3) demonstrated selective infectivity toward endometrial cells while sparing ovarian tissue. AAV3-mediated delivery of small interfering RNA targeting estrogen receptor 2 reduced Estrogen receptor beta (ERβ) expr

SUBMITTER: Baek JK 

PROVIDER: S-EPMC12472493 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature