RNA Therapy

Therapeutic Ribozymes Repair Mutant RNAs Associated With a Variety of Diseases

Published in AIDS Weekly, June 17th, 1996

A trans-splicing group I ribozyme can be therapeutically utilized to modify the sequence of targeted RNA transcripts.

Ribozymes are RNA molecules with enzymatic activity and have great potential as therapeutic entities because of their ability to either cleave deleterious RNA transcripts or repair mutant cellular RNAs. Certain catalytic ribozymes can cut and splice RNAs inside cells, modifying the encoded genetic information.

Development of ribozymes as therapeutic agents in vivo has been hampered by the inability to directly identify and characterize ribozyme reaction products in mammalian cells. Joshua T. Jones et al. developed an...

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Source: AIDS Weekly (1996-06-17)

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