Emerging trends in gene and cell therapy: CRISPR in DNA editing and beyond
Year: 2025
Type: article
Abstract: CRISPR-based gene and cell therapies are rapidly transitioning from experimental platforms to clinical reality, exemplified by the recent approval of CRISPR-derived treatments for β-hemoglobinopathies. This review highlights how advances in genome editing technologies, ranging from CRISPR-Cas nucleases to base and prime editors, are expanding the therapeutic landscape beyond traditional gene knockout approaches. We focus on the clinical translation of these tools, drawing on examples from ongoing and completed human trials to illustrate their potential across diverse disease areas. Furthermore, we discuss critical considerations such as delivery challenges, long-term safety, immune responses, and editing specificity, all of which are critical to the safe and effective integration of CRISPR technologies into modern medicine.
Source: Cell Reports Medicine
Authors: Ruijin Ji, Qiubing Chen, Ying Zhang
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $5,620
Funders: National Natural Science Foundation of China, China Postdoctoral Science Foundation, Natural Science Foundation of Hubei Province, National Key Research and Development Program of China, Fundamental Research Funds for the Central Universities, National Key Research and Development Program of China Stem Cell and Translational Research
OpenAlex ID: W4417535790