What question did this study set out to answer?

To explain the recent advancements in gene and cell therapies and their implications for treating various diseases.

February 28, 2026Journal of Clinical Medicine0 citationsOpen Access

The New Wave of Gene and Cell Therapies Across Diseases

ARAdrianna RieskeCentrum Badań i Innowacji Pro-Akademia DGDagmara GrotMedical University of Lodz CTCezary TrędaMedical University of Lodz

Key Points

To explain the recent advancements in gene and cell therapies and their implications for treating various diseases.
Showcased a range of therapies targeting genetic and non-genetic diseases.
Emphasized the use of CAR-T and CAR-NK cells engineered with CRISPR and transgenes.
Highlighted strategies like 'off-the-shelf' therapies to reduce immune rejection.
Discussed delivery methods including lipid nanoparticles and adeno-associated virus vectors.
Notable progress in cancer treatment with modified CAR-T and CAR-NK cells.
Advancements in treating hereditary diseases like Huntington's and CPS1 deficiency.
Emerging applications in diabetes and heart diseases, utilizing tRNA gene editing methods.

Abstract

Recent years have seen rapid progress in biological treatments for genetic diseases, as well as conditions like type 1 diabetes that lack an obvious genetic component. The authors sought to explain why this progress has emerged at this particular moment. The best way to illustrate this is by showcasing a wide range of therapies targeting diverse diseases. This progress has been driven by technological advances in genetically modified CAR-T and CAR-NK cells (e.g., using CRISPR or transgenes), which have led to significant improvements in cancer therapy. A key trend now is the emergence of “off-the-shelf” approaches aimed at generating cellular therapies compatible with a range of recipients by mitigating alloreactivity and immune rejection. Different diseases impose distinct biological and logistical limitations; thus, treatment of each patient requires an appropriate strategy. Emerging advances include the modification of therapeutic cells, either ex vivo or in vivo. Current options for transgene delivery mainly comprise lipid nanoparticles (LNPs), adeno-associated virus (AAV) vectors, and lentiviral vectors. Researchers also focus on selecting suitable promoters for specific expression in selected cell types. Altogether, these advances have led to remarkable progress in treating various diseases in recent years. This publication discusses the development of biological therapies, with particular emphasis on cell and gene therapies, illustrated by viable examples across various disorders. It covers implemented solutions for several types of cancer, as well as selected hereditary diseases and syndromes, including Huntington’s disease, carbamoyl phosphate synthetase 1 (CPS1) deficiency, hemiplegia, epidermolysis bullosa, chronic granulomatous disease, and congenital deafness. Emerging applications in heart diseases and diabetes are also summarized, along with therapeutic strategies involving tRNA gene editing. Although numerous strategies exist, only the most representative, practical, and up-to-date examples are emphasized.

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