[Synthetic oligonucleotides and their application in gene therapy].
Synthetic oligonucleotides are useful for research in molecular biology, for clinical diagnosis and for the development of new therapeutic agents. In fact, their application to gene therapy has given rise to a new field termed antisense, enabling the synthesis of a new generation of drugs. The procedure is based on coupling an oligonucleotide with mRNA to yield the protein whose production by the host is to be prevented, and thus through diverse pathways inhibiting gene expression. In medicine, a start has been made by applying antisense techniques in human drug trials, particularly as antiviral and antileukemic agents.
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Istituto di Richerche di Biologia Molecolare, Roma, Italia.
D000818:Animals; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D051379:Mice; D016376:Oligonucleotides, Antisense / Q000737:chemistry / Q000627:therapeutic use*
D016376:Oligonucleotides, Antisense
Oligonucleótidos sintéticos y su aplicación en terapia génica.