One Small Company that's Close to Curing Genetic Disease

Ed. Note: Yesterday the FREE Tomorrow in Review e-letter continued its monthly “7 Things You Need to Know” series with the second insightful contribution from scientific and financial journalist Stephen Petranek. (In case you missed it, you can read it for yourself, right here.) Today, he returns with the next two installments of his series. Read on…

4. Our Increasingly Vulnerable Computers

A Russian cybertheft ring has grabbed more than a billion online username and password combinations, including 500 million email addresses.

There’s no telling at this point whose personal credentials have been grabbed.

Security experts say the thefts came from relatively unprotected venues such as recipe and auto repair websites.

Bank account sites and other high financial risk sites should have been able to refute the attack.

Nonetheless, if you haven’t updated your passwords recently, this is a call to action.

USB Flash DriveThe state-sponsored Stuxnet worm, which infiltrated Iran’s heavily fortified Natanz nuclear facility and spread to countless other machines connected to the Internet, used a simple USB stick. “It turns out there is always an idiot around who doesn’t think much about the thumb drive in their hand,” said one of the program’s architects.

  • At least one cyber specialist now says that in-flight Internet access systems can be hacked. If so, anything on your computer is vulnerable while using such a system aloft.
  • Beware of what you connect to your computer via USB. New software called BadUSB shows that a USB device’s control software, such as a USB drive or mouse, can be used to take control of a computer and make it perform any of a wide variety of malicious tasks. The problem isn’t easy to correct, because it’s built into the way a USB connector works.

5. Curing Genetic Diseases Is Not Far-Out

One small biotech has been making a lot of news over a possible therapy for a particularly bad form of muscular dystrophy, called Duchenne muscular dystrophy (DMD).

If you’ve followed us on this story for a while, you know this is a very serious genetic disease that impacts a gene coding for the largest protein in the body, dystrophin.

In these patients, the gene is damaged, so the protein isn’t produced — and without that protein, muscles waste away, eventually causing death. In Duchenne, the gene is sex-linked, so in almost all cases, only affects boys.

Duchenne Muscular DystrophyA genetic cure for DMD would enable the lame to walk again; already, Sarepta Therapeutics has increased the lifespans of those affected by DMD

The little biotech working on a therapy, Sarepta Therapeutics, hopes to halt the deadly disease by patching how the gene is read by courier proteins — messenger RNAs — inside the cell responsible for carrying the code to the protein manufacturing centers.

And so far, results look very good. But the downside to this therapy is a lifetime of very expensive injections. Of course, this still represents a tremendous leap forward in treating the disease.

New technologies are on the horizon, however, that hold the promise to cure this disease — and other genetic diseases — for good. Instead of working with RNAs, these new methods work by rewriting the genes themselves.

Researchers at the University of Texas Southwestern Medical Center in Dallas are working on doing just that using a genome editing system called CRISPR/Cas9.

CRISPR is a bacterial immune system, and Cas9 is an enzyme that can be guided to modify DNA at any desired location on the genome. Bacteria use it to adapt and defend against viral attacks by modifying their own DNA as a defensive mechanism.

But researchers have recently discovered how to use this molecule to modify DNA and possibly find a way to rewrite bad genes.

The UT Southwestern Medical Center researchers were able to take a mouse model of DMD and correct the mutation responsible for it. Furthermore, an improvement in the health of the mice was observed, even though many of the cells didn’t have their genes corrected.

The big challenge of these therapies is getting the gene-editing material into more cells.

In the future, we’ll use technology similar to what’s being worked on in Dallas to permanently cure diseases like DMD — not just DMD, but a whole range of genetic diseases.

Ad lucrum per scientia (toward wealth through science),

Stephen Petranek and Ray Blanco
for The Daily Reckoning

Ed. Note: Readers of the Tomorrow in Review FREE e-letter received follow-up advice on the analysis of Sarepta Therapeutics. They also received an actionable investment idea on how to invest in the next age of cybersecurity and even the next phase of the entire Internet itself! You can’t afford to miss another issue. Sign up for the Tomorrow in Review FREE e-letter here.