Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy

CRISPR-Cas-based Human Disease Therapy is a comprehensive volume that covers CRISPR-Cas9-based genome editing in mammalian cells, including disease modeling, cancer therapy, neurological disorders, heredity, blood disorders, gene correction, stem cells therapy, epigenetic modifications, patents, ethics, biosafety, and regulatory issues. It is a valuable resource for beginners and experts in the field.

Format: Hardback
Length: 390 pages
Publication date: 15 June 2021
Publisher: Elsevier Science & Technology

Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy is a comprehensive compilation of chapters authored by esteemed scientists from across the globe. CRISPR-Cas9 stands as a pivotal technology for precise genome editing and regulation in various organisms, including mammalian cells. It offers a rapid, straightforward, and cost-effective approach. The CRISPR-Cas system has recently garnered significant scientific and public attention. This volume delves into CRISPR-Cas9-based mammalian genome editing, disease modeling, cancer therapy, neurological disorders, heredity, blood disorders, defective gene correction, stem cell therapy, epigenetic modifications, patents, ethics, biosafety, and regulatory issues. Challenges and opportunities abound in this field. This book serves as a valuable resource for beginners in mammalian genome editing, as well as students, researchers, scientists, policymakers, clinicians, and stakeholders engaged in genome editing across various domains.

Introduction:
The CRISPR-Cas system, derived from the bacterial defense mechanism known as CRISPR-Cas9, has revolutionized the field of genome editing. It allows for precise and targeted modification of DNA sequences, offering immense potential for treating human diseases. In this book, we will explore the applications of CRISPR-Cas-based therapy in various disease areas, including cancer, neurological disorders, and genetic diseases.

Cancer Therapy:
Cancer is a complex and multifactorial disease characterized by the abnormal growth and spread of cells. Traditional cancer treatments such as chemotherapy and radiation often have severe side effects and are not always effective. CRISPR-Cas-based therapy has shown promising results in treating cancer cells by targeting specific genes involved in tumor growth and metastasis. One of the most promising approaches is CRISPR-Cas9-mediated gene knockout, which involves the deletion of cancer-promoting genes. Studies have shown that CRISPR-Cas9 can effectively kill cancer cells in vitro and in vivo, with minimal side effects. Another approach is CRISPR-Cas9-mediated gene editing, which involves the correction of genetic mutations that contribute to cancer development. This can help restore the function of tumor suppressor genes or inhibit the growth of cancer cells.

Neurological Disorders:
Neurological disorders are a wide range of conditions that affect the brain, spinal cord, and nerves. CRISPR-Cas-based therapy has the potential to treat these disorders by targeting specific genes or proteins involved in the development and progression of the disease. For example, researchers are exploring the use of CRISPR-Cas9 to correct genetic mutations that cause Huntington's disease, a neurodegenerative disorder that causes progressive cognitive decline and motor symptoms. Another approach is CRISPR-Cas9-mediated gene editing to enhance the expression of genes that are involved in repairing damaged nerve cells or promoting neuroplasticity, which is the ability of the brain to adapt and reorganize itself.

Genetic Diseases:
Genetic diseases are disorders caused by mutations in genes that regulate the function of cells or tissues. CRISPR-Cas-based therapy has the potential to treat these diseases by correcting or modifying the genetic mutations that cause the disease. For example, researchers are using CRISPR-Cas9 to correct genetic mutations that cause cystic fibrosis, a genetic disorder that affects the lungs, pancreas, and other organs. Another approach is CRISPR-Cas9-mediated gene editing to enhance the expression of genes that are involved in repairing damaged tissues or promoting tissue regeneration.

Ethics and Regulatory Issues:
The use of CRISPR-Cas-based therapy raises ethical and regulatory issues that need to be carefully considered. One of the main concerns is the potential for unintended consequences or off-target effects. CRISPR-Cas9 is a powerful tool, and any errors in its use could have serious consequences for the patient. Therefore, rigorous testing and regulation of CRISPR-Cas-based therapies are essential to ensure their safety and effectiveness. Another issue is the accessibility of CRISPR-Cas-based therapies. While the technology is rapidly advancing, it may not be available to all patients due to cost constraints or other factors. Therefore, efforts need to be made to make CRISPR-Cas-based therapies more accessible and affordable to patients worldwide.

Conclusion:
CRISPR-Cas-based therapy has the potential to revolutionize the treatment of human diseases. By targeting specific genes or proteins involved in disease development, CRISPR-Cas-based therapies can offer precise and effective treatments with minimal side effects. However, rigorous testing and regulation are essential to ensure the safety and effectiveness of these therapies. With continued research and development, CRISPR-Cas-based therapy has the potential to improve the quality of life for millions of patients worldwide.

Weight: 740g
Dimension: 160 x 292 x 24 (mm)
ISBN-13: 9780323853231