Year: 2023
Type: editorial
Abstract: Editorial on the Research Topic Bioengineered gene and cell therapy for treating cardiovascular diseases Gene-and cell-based therapy applications show efficacy for the treatment of a variety of diseases, including cardiovascular diseases (Zhao et al., 2023).However, their therapeutic potential is currently not fully exploited due to several reasons.Firstly, delivering therapeutic cells or genetic material to the targeted site within the cardiovascular system can be challenging.It is crucial to ensure that the cells or genes reach the intended location and integrate effectively into the damaged tissues.Achieving precise targeting and efficient delivery remains a significant hurdle.Secondly, cell and gene therapies often involve the use of embryonic stem cells or gene-editing technologies.Ethical concerns related to the source of cells, manipulation of genetic material, and potential unintended consequences of gene editing need to be carefully addressed and monitored.Last but not least, ensuring long-term efficacy and durability of cell and gene therapies is a significant challenge.Transplanted cells may experience limited survival or fail to function optimally in the long run due to poor maturity.Gene editing lay the foundation for creating novel gene therapy products, and the emergence of the clustered regularly interspaced short palindromic repeats (CRISPR)/ Cas9 system has revolutionized genome editing due to its simplicity and effectiveness.The CRISPR/Cas9 system also has the inherent risk of unintended gene editing with similar DNA sequences and causes adverse effects.Of particular concern is that off-target effects might lead to the tumorigenicity of cells.Guo et al. gave an overview of how we can manage those off-target effects for better gene and cell therapy.Although multiple informative in silico prediction tools have been developed, the computational methods are usually biased towards sgRNA-dependent off-target effects that need experimental sequencing to reach consensus.Indeed, multiple sequencing methods including GUIDE-seq, Discover-seq, and Digenome-seq have been invented to quantify the off-targeting.In addition, several strategies for reducing the off-target effects are also summarized.
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $3,295
Funder: National Cancer Institute
OpenAlex ID: W4385461683