A new age of precision gene therapy

Gene therapy has become a clinical reality as market-approved advanced therapy medicinal products for the treatment of distinct monogenetic diseases and B-cell malignancies. This Therapeutic Review aims to explain how progress in genome editing technologies offers the possibility to expand both ther...

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Published inThe Lancet (British edition) Vol. 403; no. 10426; pp. 568 - 582
Main Authors Schambach, Axel, Buchholz, Christian J, Torres-Ruiz, Raul, Cichutek, Klaus, Morgan, Michael, Trapani, Ivana, Büning, Hildegard
Format Journal Article
LanguageEnglish
Published England Elsevier Ltd 10.02.2024
Elsevier Limited
Subjects
Blood diseases
Cell cycle
Clinical trials
CRISPR
CRISPR-Cas Systems
Editing
FDA approval
Gene Editing
Gene therapy
Genetic Therapy
Genome editing
Genomes
Hemophilia
Humans
Lymphocytes B
Malignancy
Mutation
Proteins
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Summary:Gene therapy has become a clinical reality as market-approved advanced therapy medicinal products for the treatment of distinct monogenetic diseases and B-cell malignancies. This Therapeutic Review aims to explain how progress in genome editing technologies offers the possibility to expand both therapeutic options and the types of diseases that will become treatable. To frame these impressive advances in the context of modern medicine, we incorporate examples from human clinical trials into our discussion on how genome editing will complement currently available strategies in gene therapy, which still mainly rely on gene addition strategies. Furthermore, safety considerations and ethical implications, including the issue of accessibility, are addressed as these crucial parameters will define the impact that gene therapy in general and genome editing in particular will have on how we treat patients in the near future.
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ISSN:0140-6736
1474-547X
1474-547X
DOI:10.1016/S0140-6736(23)01952-9