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Earning A Nobel

Scientists have long been dissatisfied with using viruses to deliver gene therapy. It's dangerous for the patient, expensive, and impossible to scale up for widespread pharmaceutical purposes. Viral vectors have been useful for experimentation, but have shown little promise in the treatment of patients.

There has to be a better way. And scientists at the University at Buffalo may have found it. Using a form of nanoengineered silicon they're calling ORMOSIL (amino-functionalized organically modified silica) these scientists delivered genes into the brains of living mice.

A key advantage of the UB team's nanoparticle is its surface functionality, which allows it to be targeted to specific cells, explained Dhruba J. Bharali, Ph.D., a co-author on the paper...

In their first experiment the UB scientists surgically injected an ORMOSIL/DNA complex that targeted the dopamine neurons within the brain.

It worked.

No toxicity associated with the ORMOSIL nanoparticles was observed four weeks after transfection and the efficiency of the transfection equaled or exceeded results with using viral vectors, they say.

Then, using a new optical fiber in vivo imaging technique (CellviZio), developed by Mauna Kea Technologies, Paris, the researchers were able to observe the brain cells expressing transfected genes without having to sacrifice the animal.

Wow. I'm simply blown away by the leap this represents. They've developed a nonviral vector that successfully delivered its DNA load to specifically targeted cells within living animals. Then, they were able to monitor the DNA load doing its job expressing genes, within that living animal. Remarkable. I would have guessed that this sort of development was a decade away.

As if this wasn't enough, the UB team did a follow-up experiment. With another tailored form of ORMOSIL, these researchers activated adult brain stem/progenitor cells within living mice brains. It was the first time that any scientists have proven that these idle adult stem cells can be activated with gene therapy (of any kind - its never been done with viral vectors). It is believed that these activated adult stem cells can replace neurons destroyed by neurodegenerative diseases.

These scientists believe that a library of ORMOSIL nanoparticles can be developed. Each form of ORMOSIL to target a different tissue.

Somebody's getting a Nobel.

If you're keeping score, this news is a big deal because:

  1. Virus vectors are dangerous. ORMOSIL did not trigger an immune response or any form of toxicity - the mice were fine a month later.

  2. Virus vectors are expensive/impossible to scale up for widespread use in patients. ORMOSIL can be easily synthesized by chemists for widespread use.

  3. ORMOSIL worked at least as efficiently as virus (and this is the first generation of this technology).

  4. This team has demonstrated an ability to hit their target, twice. And these scientists believe that a library of these molecules can be developed to target any tissue.

  5. This wasn't done in a petri dish. This was done in a living mouse.

  6. And they were even able to monitor the gene load being expressed within a living brain using CellviZio.


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Comments

I'd worry about toxicity of the remnant silicon, but this is an amazing advance.  I'm amazed Futurepundit didn't beat you to it. ;-)

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