Research progress and challenges of in vivo gene therapy
WANG Shan
WANG Qiang
LIU Yan
Abstract：Gene therapy is a major driving force for the development of medical and biological fields,providing potential curative methods for many diseases caused by genetic defects.According to the treatment approach,it can be divided into in vivo gene therapy and in vitro gene therapy.In recent years,in vivo gene therapy has attracted much attention due to its advantages such as simple operation and the ability to avoid pre-treatment toxicity.At the same time,innovative breakthroughs in various precise gene editing technologies and vector delivery technologies have further promoted the in-depth exploration and research of in vivo therapies by scientific researchers.However,currently,most in vivo therapies are still in the pre-clinical research stage,and the translation from mouse research to human research still faces many challenges.This review is committed to partially sorting out the frontier research progress in the field of in vivo gene therapy at home and abroad and deeply analyzing the opportunities and challenges it faces from multiple dimensions.
Keywords：gene therapy in vivogene editingdelivery systemtargeted therapychallenge
Publication Date：2025-10-30
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：10( 2141-2150 )