Smart nanomaterials are transforming advanced cell engineering, cellular therapy, and regenerative medicine by enabling the highly targeted delivery of diverse therapeutic agents. These platforms are designed to efficiently and safely transport biologics, including proteins, nucleic acids, and engineered mRNA, directly into living cells. This is a major breakthrough for gene editing, where precise delivery of Cas9 components can correct mutations responsible for rare diseases.
In cancer immunotherapy, nanomaterials are enhancing the creation and effectiveness of CAR-T cells, leading to more efficient cell modification and improved anticancer activity. Innovations in nanomaterial-enabled mRNA delivery are also accelerating the development of personalized therapies, from protein replacement strategies to transient gene expression techniques. The versatility and tunability of smart nanomaterials allow them to adapt to complex biological environments, supporting precise control over cellular processes in both space and time. As a result, these technologies are opening new opportunities to treat genetic disorders, cancer, and other medical conditions that have been difficult to address using traditional methods.
The main goal of this Research Topic is to tackle key challenges that limit the clinical application of advanced therapeutics in cell engineering, gene editing, and regenerative medicine. Technologies like CRISPR-Cas9 gene editing, CAR-T cell therapy, and mRNA-based treatments show promise. However, their practical use is often restricted by poor intracellular delivery, off-target effects, and immune-related side effects. Smart nanomaterials present a powerful solution, offering precise, minimally invasive, and easily adjustable platforms for targeted transport of proteins, nucleic acids, and therapeutic mRNA into specific cell populations. This research aims to optimize nanomaterial systems for controlled delivery of Cas9 gene editors, improve the production and function of CAR-T cells for cancer treatment, and enhance direct delivery of engineered mRNA and other biologics for personalized therapy.
By innovating nanomaterial engineering and application, we hope to overcome delivery challenges, reduce off-target responses, and realize the full potential of new therapeutic approaches. Ultimately, these efforts will support the development of safer, more effective, and accessible treatments for complex medical conditions like rare genetic disorders and cancer.
Key themes for this Research Topic include but are not limited to the following:
• Innovative nanomaterial platforms for efficient and safe delivery of therapeutic agents, such as Cas9, mRNA, and biologics • Strategies for cell engineering using nanotechnology • Advances in RNA engineering • Improvements in CAR-T cell and other cell-based therapies that use nanomaterials • New approaches for treating rare diseases and cancer • Methods for overcoming barriers to clinical translation
We welcome Original Research articles, Reviews, Methods, and Perspectives. Submissions are encouraged from multidisciplinary teams working at the intersection of nanotechnology, molecular biology, engineering, and translational medicine.
Topic Editor Dr. Salman Mustfa holds shares in AstraZeneca. Topic Editor Dr. James Button holds shares in AstraZeneca and Maravai. The other Topic Editors report no competing interests related to this Research Topic.
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