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Expert Opinion on Investigational Drugs Volume 9, 2000 - Issue 5
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Review

Therapeutic potential of ribozymes in haematological disorders

Helen A James School of Biological Sciences, University of East Anglia, Norwich, Norfolk, NR4 7TJ, UK. [email protected]
Pages 1009-1020 | Published online: 24 Feb 2005

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  • ••The first description of a potentially very important class ofribozymes, the trans-splicing ribozymes.
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  • ••The description of a trans-cleaving hammerhead ribozymethat led to the development of potentially clinically useful ribozymes.
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  • •A clever idea: designing the ribozyme to cleave at the ras activating mutation.
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Websites

  • •Report of the first antisense molecule to make it to the clinic.
  • AMADO R, ROSENBLATT J, ZACK J et al.: A Phase I gene therapy trial of autologous CD34+ cells transduced with an anti-HIV ribozyme. 6th Conference on retrovE ruses and opportunistic infections. (1999) http://www.retroconference.org/99/abstracts/17.htm
  • http://www.rpi.com/heptazyme.cfm RIBOZYME PHARMACEUTICALS INC: HEPTAZY Mr.
  • http://www.rpi.com/clinical.cfm (12/4/2000) Discontinuation of clinical trials by RPI and Chiron.

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