SAB NewsThe Promise of Gene-Specific TherapiesPosted 4/13/2004 RNA interference or RNAi is a cellular mechanism that can be used to regulate gene expression and viral replication. Double-stranded, small interfering RNA molecules known as siRNA mediate this mechanism. RNAi holds promise for developing a brand new class of drugs that are capable of specifically turning off disease-causing genes. Of the 2,351 participants in a recent Instant Poll conducted by The Science Advisory Board, 52% believe that developing effective delivery methods will be the limiting factor in implementing RNAi-based gene therapy. An additional 29% of respondents were concerned about the functionality of such innovative therapeutics. Only 18% were concerned about the toxic effects of a new class of RNAi drugs. RNAi’s therapeutic potential is far reaching and includes the treatment of viral infections, cancers and inherited genetic disorders. However, as indicated by this poll, drug delivery remains a major technical challenge. Injecting double-stranded RNAs into patients is not feasible because nucleases will destroy the majority of the drug, and cellular membranes will inhibit the remaining RNA molecules. Even if this delivery obstacle can be overcome, very little is yet known about possible side effects of RNAi-based therapy. "We don't really understand enough about the complex molecular and cellular processes that are involved in RNAi," says Tamara Zemlo, Ph.D., MPH, Director of Scientific and Medical Communications of The Science Advisory Board. "I worry that if the field moves too fast, normal cellular pathways could be inadvertently interrupted resulting in deleterious side effects." |
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