. siRNA-directed inhibition of HIV-1 infection. Nat Med. 2002 Jul;8(7):681-6. PubMed.


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  1. This is a very straightforward paper describing the use of siRNAs as a potential therapeutic reagent against HIV infection (as does Lee et al. 2002, published last month in Nature Biotechnology). It is uncertain if targeted gene silencing using siRNA can be employed to treat neurodegenerative diseases. Efficient delivery into the neuron will be a major limitation for siRNA-mediated gene targeting in brain. However, if siRNA can be delivered selectively to the desired population of neurons, this approach could be used to inhibit the targeted protein locally, for example BACE in AD.

    I think the siRNA approach will be more widely used to create model systems in the neurodegenerative disease research field. siRNA will be particularly useful either to assess the normal function of disease-related genes (e.g. presenilin, nicastrin, and AbPP) by conveniently inducing knock-out phenotypes, or to create a model system for diseases where "loss-of-function" of the target gene product is a predicted cause of the disease, for example parkin in PD.—Tae-Wan Kim, Columbia University, New York


    . Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat Biotechnol. 2002 May;20(5):500-5. PubMed.

    . Effective expression of small interfering RNA in human cells. Nat Biotechnol. 2002 May;20(5):505-8. PubMed.

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  1. Using RNAi to Silence HIV—What about Alzheimer's?