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Synthetic biology is a combination of various elements of biology and engineering. Synthetic biology first occurred in bacterial cells, specifically E. coli, which could be genetically engineered to be resistant to kanamycin. There are two general classes of synthetic biology. The first approach does not utilize repeatable components from biological systems, but instead modifies other parts to adjust specific processes. The second one attempts to mimic living systems using artificial molecules, especially building blocks. With the development of this field over the years, several tools, such as CRISPR-Cas9, used to process genes in organisms have been well studied. Essentially, it is similar to a switch that can be used to “turn on or off” a gene. Building blocks can be used to fabricate genetic circuits. Genetic disorders can be nearly impossible to treat with just a few synthetic biological components, such as drugs, genetic processing techniques, and oscillators; thus, gene switches can serve as effective strategies for disease therapeutics. Applications of CRISPR-Cas9 by targeting faulty genes can cure diseases such as Huntington’s disease, Sickle cell disease, Hemophilia, Muscular dystrophy, and many others. Also, the use of antisense oligonucleotide (ASO) and Aden-associated virus (AAV) for gene editing has opened a new way to cure several diseases. While some genetic diseases can be cured using these tools, research to heal others is still ongoing. In recent times, synthetic biology has shown great potential for disease prevention, diagnosis, and treatment for reducing future mortality and morbidity outcomes. In this book chapter, authors discuss the potential for synthetic biology to contribute to the advancement of medical and pharmaceutical research. Moreover, authors also discuss the timeline of advancements in synthetic biology, exploring both historical and contemporary aspects related to different industrial processes, bioengineering of cells containing synthetic DNA circuits for disease diagnostics and treatments, and aspects of cell-free synthetic biology in medical and pharmaceutical applications.

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Synthetic Biology for Diagnosis and Therapy of Genetic Disorders

  • 工具书词条

  • 发表日期：

    2026年

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  • Devangi Zala,
  • Shuvomoy Banerjee,
  • Ansh Mehta,
  • Anand K. Tiwari

摘要

Synthetic biology is a combination of various elements of biology and engineering. Synthetic biology first occurred in bacterial cells, specifically E. coli, which could be genetically engineered to be resistant to kanamycin. There are two general classes of synthetic biology. The first approach does not utilize repeatable components from biological systems, but instead modifies other parts to adjust specific processes. The second one attempts to mimic living systems using artificial molecules, especially building blocks. With the development of this field over the years, several tools, such as CRISPR-Cas9, used to process genes in organisms have been well studied. Essentially, it is similar to a switch that can be used to “turn on or off” a gene. Building blocks can be used to fabricate genetic circuits. Genetic disorders can be nearly impossible to treat with just a few synthetic biological components, such as drugs, genetic processing techniques, and oscillators; thus, gene switches can serve as effective strategies for disease therapeutics. Applications of CRISPR-Cas9 by targeting faulty genes can cure diseases such as Huntington’s disease, Sickle cell disease, Hemophilia, Muscular dystrophy, and many others. Also, the use of antisense oligonucleotide (ASO) and Aden-associated virus (AAV) for gene editing has opened a new way to cure several diseases. While some genetic diseases can be cured using these tools, research to heal others is still ongoing. In recent times, synthetic biology has shown great potential for disease prevention, diagnosis, and treatment for reducing future mortality and morbidity outcomes. In this book chapter, authors discuss the potential for synthetic biology to contribute to the advancement of medical and pharmaceutical research. Moreover, authors also discuss the timeline of advancements in synthetic biology, exploring both historical and contemporary aspects related to different industrial processes, bioengineering of cells containing synthetic DNA circuits for disease diagnostics and treatments, and aspects of cell-free synthetic biology in medical and pharmaceutical applications.