Promising gene therapy delivers treatment directly to brain

Huge advancements have been achieved in the last few years thanks to new and improved technologies, children that until now were doomed to a short, miserable life now have a chance to live a much more fulfilling and productive life even if not a completely normal one. It is difficult to understand how much this means to their families.

Risk, difficulties, huge costs remains, but going from "impossible" to "very difficult and risky" is a huge improvement, if things continue as right now for a few more years many other genetic diseases could get a cure soon and the current ones would be treated much more economically and easily. One very important point of this article is that the example of AADC deficiency is considered an "ultrarate" health problem, that makes absolutely no economic sense to cure for a company, but that still benefited from the research being done. This is part of a relatively new trend in gene and cell therapy research that is focusing on personalized treatments, so development of treatments is no longer something prioritized for relatively common diseases but instead as a general process that can be tailor fitted to patients that have problems because of a single gene mutation.

This means that instead of trying to commercialize one single variety of gene therapy companies would have the incentive to commercialize the process of the treatment so it could be used on any of many different diseases, no need to plan for a few dozen patients a year but for thousands.

Awesome.

Too expensive to be practical at this stage.