Antisense therapy

Antisense therapy

Antisense therapy is a form of treatment for genetic disorders or infections.When the genetic sequence of a particular gene is known to be causative of a particular disease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical analogue) that will bind to the messenger RNA (mRNA) produced by that gene and inactivate it, effectively turning that gene "off". This is because mRNA has to be single stranded for it to be translated. Alternatively, the strand might be targeted to bind a splicing site on pre-mRNA and modify the exon content of an mRNA [cite journal | last = Morcos | first = PA | year = 2007 | title = Achieving targeted and quantifiable alteration of mRNA splicing with Morpholino oligos | journal = Biochem Biophys Res Commun | volume = 358 | issue = 2 | pages = 521–7 | pmid = 17493584 |doi=10.1016/j.bbrc.2007.04.172] .

This synthesized nucleic acid is termed an "anti-sense" oligonucleotide because its base sequence is complementary to the gene's messenger RNA (mRNA), which is called the "sense" sequence (so that a sense segment of mRNA " 5'-AAGGUC-3' " would be blocked by the anti-sense mRNA segment " 3'-UUCCAG-5' ").

Antisense drugs are being researched to treat cancers (including lung cancer, colorectal carcinoma, pancreatic carcinoma, malignant glioma and malignant melanoma), diabetes, ALS, Duchenne muscular dystrophy and diseases such as asthma and arthritis with an inflammatory component. Most potential therapies have not yet produced significant clinical results, though one antisense drug, fomivirsen (marketed as Vitravene), has been approved by the US Food and Drug Administration (FDA) as a treatment for cytomegalovirus retinitis.

In early 2006, scientists studying the Ebola virus at USAMRIID announced a 75% recovery rate after infecting four rhesus monkeys and then treating them with an antisense Morpholino drug (the usual mortality rate for monkeys infected with Ebola is 100%). []

Also in 2006, German physicians reported on a dose-escalation study for the compound AP 12009 (a phosphorothioate antisense oligodeoxynucleotide specific for the mRNA of human transforming growth factor TGF-beta2) in patients with high grade gliomas. At the time of the report, the median overall survival had not been obtained and the authors hinted at a potential cure. [ [ Results of G004, a phase IIb actively controlled clinical trial with the TGF-b2 targeted compound AP 12009 for recurrent anaplastic astrocytoma - Hau et al. 24 (18 Supplement): 1566 - ASCO Meeting Abstracts ] ]


Weiss, B. (ed.): Antisense Oligodeoxynucleotides and Antisense RNA : Novel Pharmacological and Therapeutic Agents, CRC Press, Boca Raton, FL, 1997

Weiss, B., Davidkova, G., and Zhou, L-W.: Antisense RNA gene therapy for studying and modulating biological processes. Cell. Mol. Life Sci., 55:334-358, 1999

ee also

* antisense
* antisense mRNA
* Morpholino
* Peptide nucleic acid
* Locked Nucleic Acid

External links

* [ Vitravene Injection Information] , from
* [ Antisense Pharma]
* [ Antisense Pharma: Promising Phase IIb Results Of Targeted Therapy With AP 12009 In Recurrent Anaplastic Astrocytoma]

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Look at other dictionaries:

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  • Antisense RNA — (aRNA) is single stranded RNA that is complementary to a messenger RNA (mRNA) strand transcribed within a cell. Antisense RNA may be introduced into a cell to inhibit translation of a complementary mRNA by base pairing to it and physically… …   Wikipedia

  • antisense — See a. DNA, a. RNA. * * * an·ti·sense an .tī .sens, an ti adj having a sequence complementary to a segment of genetic material specif of, being, relating to, or possessing a sequence of DNA or RNA that is complementary to and pairs with a… …   Medical dictionary

  • therapy — 1. The treatment of disease or disorder by any method. SEE ALSO: therapeutics. 2. In psychiatry, and clinical psychology, a short term for psychotherapy. SEE ALSO: psychotherapy …   Medical dictionary

  • antisense — ˌ adjective Etymology: anti (I) + sense (as in nonsense herein) : having a sequence complementary to a segment of genetic material ; specifically : of, being, relating to, or possessing a sequence of DNA or RNA that is complementary to and pairs… …   Useful english dictionary

  • antisense — adjective Etymology: anti + nonsense Date: 1977 having a sequence complementary to a segment of genetic material; specifically of, being, relating to, or possessing a sequence of DNA or RNA that is complementary to and pairs with a specific… …   New Collegiate Dictionary

  • antisense gene — /æntiˈsɛns dʒin/ (say antee sens jeen) noun a piece of DNA that is complementary to an existing gene which, when inserted into the genome, locks the expression of the primary gene; used in gene therapy …   Australian English dictionary

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  • Sense (molecular biology) — In molecular biology and genetics, sense is a concept used to compare the polarity of nucleic acid molecules, such as DNA or RNA, to other nucleic acid molecules. Depending on the context within molecular biology, sense may have slightly… …   Wikipedia

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