Feeds

Lab mice deaths cast doubt over RNAi

Setback for powerful therapy?

The next step in data security

A technology that has been lauded as a panacea for many currently incurable diseases has been dealt a blow by researchers. A paper appearing in Nature says mice given RNA interference therapy (RNAi) died of liver failure and other complications.

RNA is a close chemical relative of DNA. While DNA can be thought of as an unchanging leather-bound archive, RNA molecules are more like newspapers – dynamic, throwaway and with different agendas. RNA is the workhorse of biological information.

The principle behind RNAi is to use short double-stranded tracts of RNA as guided missiles, intercepting longer RNA messages which pass on information from DNA, before they can be used to make functioning proteins. It's thought to have developed early in evolution - bacteria use it too - as a means of combating viral infections.

Teams are working on harnessing its power to treat both deadly inherited conditions like Huntington's and infectious disease, such as HIV.

For this latest study, a team at Stanford medical school exposed mice to long-term high doses of RNAi. They tried out 49 different RNAi therapies designed to shut down six different genes in liver cells. 36 of the snippets caused liver injury, and 23 led to death within two months.

For biotech firms pumping millions into the field, the news may bring concerning echoes problems researchers have had fulfilling the potential of similarly promising gene therapy technology, which gave patients cancer in an early trial. Proponents say the study should act as a warning, but does not signal disaster for RNAi.

The Stanford team used an indirect approach to introduce the RNAi – they forced the mice's own cells to build them. The scientists speculate that this may have overloaded the cellular machinery, leading to the liver damage and deaths.

John Maraganore of biotech firm Alnylam Pharmaceuticals, which says it has already safely tested a respitory infection RNAi nasal spray told the NYT: "These data really represent the fundamental limitations of gene therapy, not of RNAi."

The authors of the study also note, despite the mouse deaths, other work they did proving the ability of RNAi to silence errant genes "fuels hope" of a whole new mode of attack against illness. ®

New hybrid storage solutions

More from The Register

next story
PORTAL TO ELSEWHERE scried in small galaxy far, far away
Supermassive black hole dominates titchy star formation
Bacon-related medical breakthrough wins Ig Nobel prize
Is there ANYTHING cured pork can't do?
Boffins say they've got Lithium batteries the wrong way around
Surprises at the nano-scale mean our ideas about how they charge could be all wrong
Edge Research Lab to tackle chilly LOHAN's final test flight
Our US allies to probe potential Vulture 2 servo freeze
Europe prepares to INVADE comet: Rosetta landing site chosen
No word yet on whether backup site is labelled 'K'
Cracked it - Vulture 2 power podule fires servos for 4 HOURS
Pixhawk avionics juice issue sorted, onwards to Spaceport America
Archaeologists and robots on hunt for more Antikythera pieces
How much of the world's oldest computer can they find?
prev story

Whitepapers

Secure remote control for conventional and virtual desktops
Balancing user privacy and privileged access, in accordance with compliance frameworks and legislation. Evaluating any potential remote control choice.
WIN a very cool portable ZX Spectrum
Win a one-off portable Spectrum built by legendary hardware hacker Ben Heck
Intelligent flash storage arrays
Tegile Intelligent Storage Arrays with IntelliFlash helps IT boost storage utilization and effciency while delivering unmatched storage savings and performance.
High Performance for All
While HPC is not new, it has traditionally been seen as a specialist area – is it now geared up to meet more mainstream requirements?
Beginner's guide to SSL certificates
De-mystify the technology involved and give you the information you need to make the best decision when considering your online security options.