UCLA team has figured out how to correct two (laboratory cell line) mitochondrial DNA diseases by importing corrective RNAs — next come lab animal tests

This may be a game-changer

The University of California at Los Angeles reported that:

[P]ost-doctoral fellow Geng Wang developed a strategy to target and import specific RNA molecules encoded in the nucleus into the mitochondria and, once there, to express proteins needed to repair mitochondrial gene mutations.

First, the research team had to figure out a way to stabilize the reparative RNA so that it was transported out of the nucleus and then localized to the mitochondrial outer membrane. This was accomplished by engineering an export sequence to direct the RNA to the mitochondrion.

Once the RNA was in the vicinity of the transport machinery on the mitochondrial surface, then a second transport sequence was required to direct the RNA into the targeted organelle.

With these two modifications, a broad spectrum of RNAs were targeted to and imported into the mitochondria, where they functioned to repair defects in mitochondrial respiration and energy production in two different cell line models of human mitochondrial disease.

© 2012 Newswise, Correcting Human Mitochondrial Mutations, University of California – Los Angeles Health System (12 March 2012) (paragraph split)

Citation

Geng Wang, Eriko Shimada, Jin Zhang, Jason S. Hong, Geoffrey M. Smith, Michael A. Teitell, and Carla M. Koehler, Correcting human mitochondrial mutations with targeted RNA import, Proceedings of the National Academy of Sciences [PNAS], doi: 10.1073/pnas.1116792109 (published online before print, 12 March 2012)

If this works, it will be a really big deal

Early-on promises for the virtues of reparative genetic engineering have not been met.  If Wang et al.’s technique works in animals and humans, medicine may finally be into a new era.