Gene-and cell-based therapy applications show promise for the treatment of a variety of diseases, including cardiovascular diseases. However, the therapeutic potential is currently not fully realized. On one hand, genome editing has the inherent risk of unintended editing of genes with similar DNA sequences. Of particular concern is that off-target effects can lead to tumorigenicity of cells, and imprecise delivery of gene therapy products into unintended cells further aggravates the condition. On the other hand, technological hurdles block the way to generating purified therapeutic products efficiently. The contaminants could severely compromise the efficacy by eliciting unwanted responses. Last but not least, our body's immune system might react to the newly introduced therapeutics as if it were an intruder. A reaction of the immune system may lead to inflammation and other serious risks.
This research topic aims to highlight recent advances in bioengineered next-generation gene and cell therapy for modeling and treating cardiovascular diseases. As a multidisciplinary field, crosstalk between science, engineering, and clinic contributed to new products ranging from physiologically relevant disease models to specifically targeted delivery vehicles. For example, in vitro cardiovascular organoids in combination with microphysiological systems provide an excellent platform for preclinical testing. Novel organ-selective targeting lipid nanoparticles (LNPs) facilitate the delivery of gene products such as siRNA and modified mRNA. In addition, synthetic biology could inject the logic circuit into the stem cells and further program stem cells into fail-safe therapeutic cells much more efficiently compared to traditional differentiation.
We welcome contributions in the form of original research articles, review articles, mini-reviews, and perspectives on novel technologies for advancing gene- and cell-based therapy from the laboratory to the bedside. These include, but are not limited to:
1. Cell therapy
2. Stem cell and organoids
3. Genome and Epigenome editing
4. Gene therapy (viral and non-viral)
5. Gene-, and cellular delivery systems
Please note that clinical studies and manuscripts describing purely bioinformatic analyses of existing databases without significant experimental validation will not be accepted into this collection, as they are outside the scope of the Preclinical Cell and Gene Therapy section, of which this Research Topic is a part.
Gene-and cell-based therapy applications show promise for the treatment of a variety of diseases, including cardiovascular diseases. However, the therapeutic potential is currently not fully realized. On one hand, genome editing has the inherent risk of unintended editing of genes with similar DNA sequences. Of particular concern is that off-target effects can lead to tumorigenicity of cells, and imprecise delivery of gene therapy products into unintended cells further aggravates the condition. On the other hand, technological hurdles block the way to generating purified therapeutic products efficiently. The contaminants could severely compromise the efficacy by eliciting unwanted responses. Last but not least, our body's immune system might react to the newly introduced therapeutics as if it were an intruder. A reaction of the immune system may lead to inflammation and other serious risks.
This research topic aims to highlight recent advances in bioengineered next-generation gene and cell therapy for modeling and treating cardiovascular diseases. As a multidisciplinary field, crosstalk between science, engineering, and clinic contributed to new products ranging from physiologically relevant disease models to specifically targeted delivery vehicles. For example, in vitro cardiovascular organoids in combination with microphysiological systems provide an excellent platform for preclinical testing. Novel organ-selective targeting lipid nanoparticles (LNPs) facilitate the delivery of gene products such as siRNA and modified mRNA. In addition, synthetic biology could inject the logic circuit into the stem cells and further program stem cells into fail-safe therapeutic cells much more efficiently compared to traditional differentiation.
We welcome contributions in the form of original research articles, review articles, mini-reviews, and perspectives on novel technologies for advancing gene- and cell-based therapy from the laboratory to the bedside. These include, but are not limited to:
1. Cell therapy
2. Stem cell and organoids
3. Genome and Epigenome editing
4. Gene therapy (viral and non-viral)
5. Gene-, and cellular delivery systems
Please note that clinical studies and manuscripts describing purely bioinformatic analyses of existing databases without significant experimental validation will not be accepted into this collection, as they are outside the scope of the Preclinical Cell and Gene Therapy section, of which this Research Topic is a part.