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Gene therapy and gene correction: targets, progress, and challenges for treating human diseases

Abstract

The field of gene therapy has made significant strides over the last several decades toward the treatment of previously untreatable genetic disease. Gene therapy techniques have been aimed at mitigating disease features of recessive and dominant disorders, as well as several cancers and other diseases. While there have been numerous disease targets of gene therapy trials, only four therapies have reached FDA and/or EMA approval for clinical use. Gene correction using CRISPR-Cas9 is an extension of gene therapy that has received considerable attention in recent years and boasts many possible uses beyond classical gene therapy approaches. While there is significant therapeutic potential using gene therapy and gene correction strategies, a number of hurdles remain to be overcome before they become more common in clinical use, particularly with regards to safety and efficacy. As research progresses in this exciting field, it is likely that these therapies will become first-line treatments and will have tremendous positive impacts on the lives of patients with genetic disorders.

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Fig. 1: The use of CRISPR-Cas9 for genome editing.
Fig. 2: Regulation of gene expression using Cas9.

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Funding

This work was funded by the National Institutes of Health, grant numbers R01 EY011298 (VCS), R01 EY017168 (VCS) and an institutional grant to the Department of Ophthalmology and Visual Sciences at the University of Iowa (P30EY025580), as well as the Roy J. Carver Charitable Trust (VCS).

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Cring, M.R., Sheffield, V.C. Gene therapy and gene correction: targets, progress, and challenges for treating human diseases. Gene Ther 29, 3–12 (2022). https://doi.org/10.1038/s41434-020-00197-8

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