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-Application of gene therapy in clinical trials according to Wiley Gene Therapy Clinical Trial Databases (Ginn et al., 2018). Almost 70% of all clinical trials are designed for cancer diseases (light green), while monogenic diseases (coral) account for approximately 11%.
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There are many clinical trials underway for the development of gene therapies, and some have resulted in gene therapy products being commercially approved already. Significant progress was made to develop safer and more effective strategies to deliver and regulate genetic products. An unsolved aspect is the immune system, which can affect the effic...
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Context 1
... therapy has gained momentum for treating different diseases worldwide with several clinical trials underway ( Figure 3) and has made its way to the market with over ten approved products (Table 2). However, it is interesting to note that even though the definition of gene therapy refers ...
Citations
... The immune system's identification of these viral vectors as intruders might trigger a defensive reaction, potentially neutralizing the intended therapeutic impact and inducing inflammatory reactions [317]. Moreover, the expression of therapeutic genes or proteins could trigger inflammatory responses, culminating in tissue impairment or untoward reactions [318]. Assuring the prolonged safety of gene therapy constitutes a formidable task. ...
Cardiac regeneration is a critical endeavor in the treatment of heart diseases, aimed at repairing and enhancing the structure and function of damaged myocardium. This review offers a comprehensive overview of current advancements and strategies in cardiac regeneration, with a specific focus on regenerative medicine and tissue engineering-based approaches. Stem cell-based therapies, which involve the utilization of adult stem cells and pluripotent stem cells hold immense potential for replenishing lost cardiomyocytes and facilitating cardiac tissue repair and regenera-tion. Tissue engineering also plays a prominent role employing synthetic or natural biomaterials, engineering cardiac patches and grafts with suitable properties, and fabricating upscale bioreactors to create functional constructs for cardiac recovery. These constructs can be transplanted into the heart to provide mechanical support and facilitate tissue healing. Additionally, the production of organoids and chips that accurately replicate the structure and function of the whole organ is an area of extensive research. Despite significant progress, several challenges persist in the field of cardiac regeneration. These include enhancing cell survival and engraftment, achieving proper vascularization, and ensuring the long-term functionality of engineered constructs. Overcoming these obstacles and offering effective therapies to restore cardiac function could improve the quality of life for individuals with heart diseases.
... To avoid unfavorable immune responses, immunosuppressive drugs are often co-administered, a strategy employed by clinical trials using CRISPR (Gillmore et al., 2021), other gene therapy approaches (Corti et al., 2017;Mendell et al., 2017;Russell et al., 2017;Freitas et al., 2022), or cellular therapies (Ramzy et al., 2021;Shapiro et al., 2021). Corticosteroids are approved for use with two AAV-based gene therapy drugs in clinical use, Zolgensma (Mendell et al., 2017) and Luxturna (Russell et al., 2017). ...
CRISPR offers new hope for many patients and promises to transform the way we think of future therapies. Ensuring safety of CRISPR therapeutics is a top priority for clinical translation and specific recommendations have been recently released by the FDA. Rapid progress in the preclinical and clinical development of CRISPR therapeutics leverages years of experience with gene therapy successes and failures. Adverse events due to immunogenicity have been a major setback that has impacted the field of gene therapy. As several in vivo CRISPR clinical trials make progress, the challenge of immunogenicity remains a significant roadblock to the clinical availability and utility of CRISPR therapeutics. In this review, we examine what is currently known about the immunogenicity of CRISPR therapeutics and discuss several considerations to mitigate immunogenicity for the design of safe and clinically translatable CRISPR therapeutics.
