Preclinical Study on the Long-Term Efficacy and Safety of Sustained-Release Gas6 Liposomes Combined With MERTK Gene Therapy for Retinitis Pigmentosa.

ABSTRACT:

Purpose

Our previous studies in Royal College of Surgeons (RCS) rats demonstrated that co-administration of MER proto-oncogene tyrosine kinase (MERTK) and its ligand Gas6 effectively protected photoreceptors and improved visual function. To facilitate clinical translation, we developed Gas6-loaded sustained-release liposomes (Gas6 Lips) using liposomes with superior biocompatibility and enhanced release stability as the delivery vehicle, and subsequently evaluated their long-term efficacy and safety in RCS rats and nonhuman primates.

Methods

Gas6 Lips were synthesized using a thin-film hydration method and characterized for morphology, encapsulation efficiency, and release profile. A GMP-grade adeno-associated virus serotype 2 (AAV2)-BEST1-hMERTK vector was constructed. Expe

SUBMITTER: Wu S 

PROVIDER: S-EPMC12710778 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature