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Advances in viral and nonviral delivery systems for therapeutic gene editing

Year: 2025

Type: article

Abstract: Genetic diseases seriously threaten human health and remain among the most challenging diseases to manage. Gene editing offers transformative potential for the treatment of genetic disorders. Over the past four decades, gene therapy has evolved significantly, with in vivo gene editing emerging as a promising strategy to correct the root causes of many hereditary conditions. Achieving therapeutic efficacy, however, depends on the development of safe and efficient delivery systems capable of protecting gene editing agents from degradation and directing them precisely to target organs, cells, and even subcellular compartments. In this review, we summarize the current delivery platforms used in vivo gene editing, including both viral and nonviral vectors. Given that no single delivery approach is universally optimal, we compare the strengths and limitations of each method and discuss their applications in treating genetic diseases.

Source: Taiwan Journal of Ophthalmology

DOI: 10.4103/tjo.tjo-d-25-00068

Authors: Weiyan Jiang, Hyun Chul Jeong, Jeong Hun Kim

Institutions: Seoul National University, Seoul National University Hospital, Seoul National University Hospital Biomedical Research Institute

Language: English

Topic: RNA Interference and Gene Delivery

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: diamond

OpenAlex ID: W4413817733

FWCI:

Cites: 69

Cited by:

Related to: 10