Precision Medicine will need to get out of the pharma silo that is based on symptoms


Welcome to the digital era of biology (and to this modest blog I started in early 2005).

To cure many diseases, like cancer or cystic fibrosis, we will need to target genes (mutations, for ex.), not organs! I am convinced that the future of replacement medicine (organ transplant) is genomics (the science of the human genome). In 10 years we will be replacing (modifying) genes; not organs!


Anticipating the $100 genome era and the P4™ medicine revolution. P4 Medicine (Predictive, Personalized, Preventive, & Participatory): Catalyzing a Revolution from Reactive to Proactive Medicine.


After low-cost airlines (Ryanair, Easyjet ...) comes "low-cost" participatory medicine. Some of my readers have recently christened this long-lasting, clumsy attempt at e-writing of mine "THE LOW-COSTE INNOVATION BLOG". I am an
early adopter of scientific MOOCs. My name's Catherine Coste. I've earned myself four MIT digital diplomas: 7.00x, 7.28x1, 7.28.x2 and 7QBWx. Instructor of 7.00x: Eric Lander PhD.

Upcoming books: Doomsdare, a medical thriller (action taking place in Beijing) Fall 2016; Jesus CRISPR Superstar, a sci-fi -- French title: La Passion du CRISPR (2017). Special thanks to Prof. Emmanuel Lincot, lawyer David Kilgour and Isabelle Provost for their help.

I love Genomics. Would you rather donate your data, or... your vital organs?

Audio files on this blog are Windows files ; if you have a Mac, you might want to use VLC (http://www.videolan.org) to read them.

Concernant les fichiers son ou audio (audio files) sur ce blog : ce sont des fichiers Windows ; pour les lire sur Mac, il faut les ouvrir avec VLC (http://www.videolan.org).


Lowering LDL cholesterol: Will gene therapy become the next Eldorado (exit Statins)?

http://www.nature.com/news/genetics-a-gene-of-rare-effect-1.12773
#CDoM About 35 million PPLE in the US (give or take) are on statins... Will monoclonal antibody to PCSK9 gene replace statins?...

Effect of a Monoclonal Antibody to PCSK9 on LDL Cholesterol

A gene called PCSK9 causes degradation of the LDL cholesterol receptor (Low Density Cholesterol is the bad guy, the one that puts us at risk for a heart attack). An LDL receptor that is properly working allows us to decrease our LDL cholesterol levels.

If this receptor is being degraded, our LDL cholesterol levels will increase (bad news).

"So, how about the complete loss of PCSK9? Turns out that it's not lethal. That's a really important thing. So when drug companies found out that you could actually knock this thing out, and it would be perfectly compatible with life, then that said, this isn't such a risky thing. And a number of companies have made antibodies against PCSK9, and they've been in clinical testing. And here's an example of the effect of a monoclonal antibody to PCSK9 on LDL cholesterol. One injection. That's it, as the antibody then washes out. Decreases quite substantially. Several companies have done this. These haven't yet been approved. I think it's very likely they will be approved. Cool. (...) There are some drugs in clinical testing right now that block that transfer of cholesterol ester from the HDLs (High Density Cholesterol, the good form of cholesterol, that is) to LDLs, and they have a dual effect. They increase HDL and simultaneously lower LDL. They may be very effective, because they're lowering the LDL."

Eric Lander PhD, Geneticist. MITx 7.00x "The Secret of Life", June 2013.

http://www.pace-cme.org/d/224/antibodies-against-pcsk9-a-new-ldl-c-lowering-therapy

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