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From Nucleic Acids Sequences to Molecular Medicine pp 19–45Cite as

Nucleic Acids as Therapeutics

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Part of the book series: RNA Technologies ((RNATECHN))

Abstract

Gene therapy is a technique for correcting defective genes responsible for disease development. Nucleic acid-based molecules (deoxyribonucleic acid, complementary deoxyribonucleic acid, complete genes, ribonucleic acid, and oligonucleotides) are utilized as research tools within the broad borders of gene therapy and the emerging field of molecular medicine. Although most of the nucleic acid-based drugs are in early stages of clinical trials, these classes of compounds have emerged in recent years to yield extremely promising candidates for drug therapy to a wide range of diseases, including cancer, infectious diseases, diabetes, cardiovascular, inflammatory, and neurodegenerative diseases, cystic fibrosis, hemophilia, and other genetic disorders. Gene therapy may be classified into two types: somatic and germ line gene therapy. There are many ethical, social, and commercial issues raised by the prospects of treating patients using gene therapy. This chapter summarizes deoxyribonucleic acid-based therapeutics, ribonucleic acid-based therapeutics, and gene transfer technologies. Deoxyribonucleic acid-based therapeutics includes plasmids, oligonucleotides for antisense and antigene applications, deoxyribonucleic acid aptamers, and deoxyribonucleic acidzymes, while ribonucleic acid-based therapeutics includes ribonucleic acid aptamers, ribonucleic acid decoys, antisense ribonucleic acid, ribozymes, small interfering ribonucleic acid, and micro ribonucleic acid. This chapter also includes current status of gene therapy and recent developments in gene therapy research.

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Authors and Affiliations

  1. Mahatma Gandhi University of Medical Sciences & Technology, RIICO Institutional Area, Sitapura, Jaipur, 302 022, India

    Saraswat Pushpendra

  2. Mahatma Gandhi Institute of Applied Sciences, Tonk Road, Sitapura, Jaipur, 302 022, India

    Pareek Arvind

  3. Jodhpur National University, Narnadi, Jhanwar Road, Jodhpur, 342 001, India

    Bhandari Anil

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  1. Saraswat Pushpendra
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Correspondence to Saraswat Pushpendra .

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  1. Institute of Chemistry/Biochemistry, Free University Berlin, Thielallee 63, Berlin, 14195, Berlin, Germany

    Volker A. Erdmann

  2. Inst. Bioorganic Chemistry, PAN Poznan, ul. Z. Noskowskiego 12/14, Poznan, 61-704, Poland

    Jan Barciszewski

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Pushpendra, S., Arvind, P., Anil, B. (2012). Nucleic Acids as Therapeutics. In: Erdmann, V., Barciszewski, J. (eds) From Nucleic Acids Sequences to Molecular Medicine. RNA Technologies. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-27426-8_2

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