and perhaps eventually, the process can be made strong enough where normal Hb levels are maintained.
Even better would be a "one-time, transformative treatment" that could provide a cure for these patients.
I think this is exactly what I would somehow call a "cure". It seems like this is also a goal that is atleast being recognized, even if it is not the main priority right now.
By allowing a patient to create his/her own blood, it eliminates the health problems related to thalassemia
Correct me if I'm wrong, but if they only reach Hb levels of "transfusion independency", this would also only free them of the serious health problems that majors have in comparison to lighter forms of thalassemia. And if we talk about a "cure" and majors could be treated without serious complications, why should we just ignore the problems of minors and everyone in between and act as if diet, exercise and other things could help us to do everything in a way a "healthy" person can do them? For me, having to build my life around thalassemia is not what I would call a "normal life". I mean, by today's standards, people with a huge variety of diseases are able to live what we call a "normal life".
Since this is only my personal feeling, I'd be curious how many minors/intermedias would do what therapy/take what medication at what "level of risk" to get rid of the problems they are experiencing.
If I go to the doctor and tell him that I'm having depressions, it's taken seriously, I'm being sent to an expert and have a large range of medication even. But I'm sure sufficient Vitamin D, a proper diet, exercise, fresh air, social interaction and other things could easily help people to fix this, while it can't simply "fix" thalassemia - only make us feel better. In contrast, if I tell a doctor that the fatigue my thalassemia causes is a trigger for my depressions and the whole thing starts on a physical level, I'm probably not taken as seriously. I guess it's just better business for all the companies, because the potential "customer base" "in need" of their medication against a wide range of mental problems that are perfectly normal and happen to everyone once in a while and are something that is not caused from a "physical suffering" is large enough to earn a ton of money.
On the general topic of gene therapy: There is something that shocked me a bit. Gene therapy seems to be extremely important to find a cure for a range of diseases.
between 2013 and April 2014, US companies invested over $600 million in gene therapy
http://en.wikipedia.org/wiki/Gene_therapySound like much for such an important area?
World's top 15 military spenders in 2013 -> USA (2013) 640.0 billion
http://en.wikipedia.org/wiki/List_of_countries_by_military_expendituresMicrosoft acquired Mojang for a smooth 2.5 BILLION dollars.
http://mojang.com/2014/09/yes-were-being-bought-by-microsoft/Facebook is acquiring WhatsApp, a company with at most $300M revenues, and 55 employees, for $19billion.
http://www.forbes.com/sites/adamhartung/2014/02/24/zuckerbergs-3-smart-leadership-lessons-from-facebook-buying-whatsapp/Snapchat Spurned $3 Billion Acquisition Offer from Facebook
http://blogs.wsj.com/digits/2013/11/13/snapchat-spurned-3-billion-acquisition-offer-from-facebook/Snapchat Inc. is near a round of funding at a valuation of about $10 billion from investors including venture capital firm Kleiner Perkins Caufield & Byers, according to a person with knowledge of the matter.
http://www.bloomberg.com/news/2014-08-26/snapchat-said-to-near-funding-with-kleiner-at-10b-value.htmlA US smartphone app that is valued as high as the amount of money spent on gene therapy research in the USA in probably decades? Wow, just wow...