the research keeps getting better - hiv, md

I previously posted the abstract of the work that could give a significant boost to our Muscular Dystrophy program. The full text is now available.

Note that in is this new publication there is also some more research on HIV and shrna.

This is all great work and, with such volumes of research going on, it cannot be long before there is a long clinical pipeline for shrna therapeutics and a long list of royalty payees to BLT.

Gee, I'm feeling good about 2012 already.

http://www.springerlink.com/content/v6v14627m7150513/about/

Here is the HIV Abstract:

Background

Combinatorial RNA interference (co-RNAi) approaches are needed to account for viral variability in treating HIV-1 with RNAi, as single short hairpin RNAs (shRNA) are rapidly rendered ineffective by resistant strains. Current work suggests that 4 simultaneously expressed shRNAs may prevent the emergence of resistant strains.

Results

In this study we assembled combinations of highly-conserved shRNAs to target as many HIV-1 strains as possible. We analyzed intersecting conservations of 10 shRNAs to find combinations with 4+ matching the maximum number of strains using 1220+ HIV-1 sequences from the Los Alamos National Laboratory (LANL). We built 26 combinations of 2 to 7 shRNAs with up to 87% coverage for all known strains and 100% coverage of clade B subtypes, and characterized their intrinsic suppressive activities in transient expression assays. We found that all combinations had high combined suppressive activities, though there were also large changes in the individual activities of the component shRNAs in our multiple expression cassette configurations.

Conclusion

By considering the intersecting conservations of shRNA combinations we have shown that it is possible to assemble combinations of 6 and 7 highly active, highly conserved shRNAs such that there is always at least 4 shRNAs within each combination covering all currently known variants of entire HIV-1 subtypes. By extension, it may be possible to combine several combinations for complete global coverage of HIV-1 variants.