Ranpirnase

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]

Overview

Ranpirnase is a ribonuclease enzyme found in the oocytes of the Northern Leopard Frog (Rana pipiens). Ranpirnase is a member of the pancreatic ribonuclease (RNase A) protein superfamily and degrades RNA substrates with a sequence preference for uracil and guanine nucleotides. Along with amphinase, another leopard frog ribonuclease, ranpirnase has been studied as a potential cancer treatment due to its unusual mechanism of cytotoxicity tested against tumor cells.[1]

Ranpirnase was originally discovered by scientists at TamirBio, a biotechnology company (formerly Alfacell Corporation), where it was tested in clinical trials under the brand name Onconase. The mechanism of action of ranpirnase tumor-selective cytotoxicity has been attributed to the RNA interference pathway, potentially through cleaving siRNA molecules;[2] to cleavage of transfer RNA;[1] and to interference with the NF-κB pathway.[3] Despite early indications of promise as a mesothelioma treatment,[4][5][6] and an orphan drug status designation from the United States Food and Drug Administration in 2007,[7] the Phase III clinical trial for this indication did not demonstrate statistical significance against primary endpoints.[8]

References

  1. 1.0 1.1 PMID 18673287 (PMID 18673287)
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  2. PMID 18927512 (PMID 18927512)
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  3. PMID 21901170 (PMID 21901170)
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  4. PMID 16305992 (PMID 16305992)
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  5. PMID 11773179 (PMID 11773179)
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  6. PMID 12860938 (PMID 12860938)
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  7. Waknine, Yael. "New FDA Orphan Drugs: Gestiva, Onconase, Aerosolized Ciprofloxacin". Medscape. Retrieved 2 February 2015.
  8. "Alfacell Annual Report 2009" (PDF). Retrieved 2 February 2015.