gene therapy

What happens when the pain gets to be too much?  What happens when the drugs stop working?  The physicians are giving you as much as you can take without dying, and youre still living with 7, 8, 9 levels of pain, every waking moment?  Some of the talk behind physician assisted suicide is to let people decide when enough is enough, and to permanently end their pain-filled hellish existence. But what if there were options? What if herpes could bring your pain levels down from 7, 8, 9... to 1-2?  What if herpes could take you from 'I want to end my own life' to 'I can handle this'? What if I…
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 lungs in textbooks, not wrapped around their childhood friends.  Diseases that once plagued childhood-- 'chicken pox', 'measles', or 'mumps'-- children today havent even heard those terms before. Vaccines are a revolution. But thats not all…
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 who need a solution the most (not just the poor abroad, I doubted the poor right here in the USA could get this kind of therapy). But in the six years Ive been writing at ERV, my stance on gene therapy has changed from 'Thats disgusting, and Im actually kind of angry…
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 considered it.  Waste of valuable resources. Flash forward to today... I am actually starting to think its not such a bad idea. Well, specific kinds of gene therapy for HIV-1 might not be such a bad idea.  One such idea is 'Chimeric Antigen Receptors', or, CARs.  CARs are half laboratory designed antibody (to…
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. Hemophilia B is a disease in males caused by point mutations/deletions/etc in the clotting Factor IX gene. If you dont make Factor IX, you wont clot properly, and will have all of the health issues we associate with hemophilia.…
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). Yes, we tend to notice them when they make us sick. But the fact of the matter is, they are super little creatures who have been evolving on this planet for millions and millions and millions of years, and have figured out how to do fantastic things that we can take advantage of for research and treatment purposes.…
Evolution connects all living things on earth, from the arsenic tolerant bacteria in the news this week to the human scientists and bloggers chatting about it. Eyes are intricately complex structures made up of many many cells, but even single-celled microbes can sense and respond to light through the function of proteins that share evolutionary similarity with the light receptors of the human retina. Incredibly, genetic engineering is showing us just how similar these proteins can be--transferring the genes that code for these processes leads to functional proteins, even when huge…
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 know this story. Some baby boys are born with a malfunctioning gene, totally screws up their immune system. The babies are fine, for a while, because they get antibodies to most things from their mothers milk. But when its time for baby to start making his own immune…
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. This was a death-blow to the HIV-1 in this guys system. The virus in him simply has no (or very few) place else to go, so it burned out. But this is simply a non-viable idea for 'curing HIV/AIDS' for basically everyone else on the planet. Financial barriers, technological barriers,…
People with red-green colour blindness find it difficult to tell red hues from green ones because of a fault in a single gene. Their inheritance robs them of one of the three types of colour-sensitive cone cells that give us colour vision. With modern technology, scientists might be able to insert a working copy of the gene into the eye of a colour-blind person, restoring full colour vision.  You might think that the brain and eye would need substantial rewiring to make use of the new hardware, but Katherine Mancuso from the University of Washington thinks otherwise. She has used gene…
A long-sought goal in genetics has been to develop therapies that can use correctly functioning genes to replace genes with defects. If we had the technology to predictably modify our genomes, we would have the ability to cure many diseases instead of having to place people on medications for their entire lives. For a long time, gene therapy has remained an elusive dream. But, in the past few years the dream has come closer to reality, especially in the case of ten children, who live because of researchers who kept that dream in sight (1). Figure 1. Random children Technorati Tags:…