Gene Therapy

Tag archives for Gene Therapy

A lot of folks have been forwarding me this story from the New York Times–  In Girl’s Last Hope, Altered Immune Cells Beat Leukemia It is a FANTASTIC story about a little girl who had been sick from leukemia for two years, wasnt responding to conventional therapies, and her parents chose to try alternative medicine. …

Lets play a game. Think of a human disease.  Any disease.  Viral, bacterial, genetic, acquired, anything. Im pretty sure that no matter what disease you just thought of, there is a scientist, somewhere, trying to use a virus to cure/treat that disease. As I was doing my rounds on PubMed, looking for cool new research,…

Gene therapy for Hemophilia A

So what do you do after you use a genetically modified virus to treat–>functionally cure Hemophilia B? Why, use a genetically modified virus to treat–>hopefully functionally cure Hemophilia A, of course! Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A Though they are both ‘hemophilia’, hemophilia…

Domesticating wild organisms– Whether its domesticating teosinte or aurochs, bending wild organisms to our will is always a game-changing event in human history. The domestication of viruses is no exception. We now live in a world where small-pox, a virus that once wiped out entire continents, is now functionally extinct.  Generations of people see iron…

GMO Virus for Nicotine Addiction

I used to think gene therapy was an absurd ‘solution’ for HIV/AIDS.  Well, ‘absurd’ is putting it lightly.  I thought gene therapy was a perverted solution– Even if it ‘worked’, it would only be available for the richest people in the richest countries, not the millions and millions and millions of individuals living in poverty…

If you had asked me 6 years ago about using gene therapy to fight HIV/AIDS, I would have given you a nice rant about how expensive it is, how non-viable it is in the places that need anti-HIV strategies the most, how dangerous it can be, and I would have been damn offended you even…

Gene therapy for Hemophilia B

For the millionth time– Viruses are not just pathogens that make us sick. In the modern world, viruses have been domesticated, and are now used to treat/cure diseases. Example #gazillion: Hemophilia B: Adenovirus-Associated Virus Vector-Mediated Gene Transfer in Hemophilia B This study is far from perfect, and of course very preliminary, but still very exciting.…

Oh, Im not a doctor.

My response: Patient: Are you sure youre a doctor? Me: Oh, Im not a doctor. If you are a regular reader of ERV, one of the Big Ideas I hope you take away from this blog is ‘Viruses are not Bad Guys‘ (more, more, this list isnt exhaustive, Ive written about this a million times).…

Yesterdays post got me so annoyed and flabbergasted– I needed to read a nice MLV paper to cheer me up. And nothing cheers me up like using gene therapy on kids with genetic diseases, allowing them to live pretty much normal lives: Efficacy of gene therapy for X-linked severe combined immunodeficiency. Most readers of ERV…

Im sure you all remember the guy I wrote about a while back, who had HIV-1 and leukemia. While that is actually pretty common, what wasnt common was his treatment– a bone marrow transplant from a match who happened to lack the CCR5 gene, thus lacked one of the co-receptors HIV-1 needs to infect cells.…