What's new in gene therapy?

Factual Questions
1

I used to study biochemistry in school about 20 years ago. There was an early experiment where a person with a genetic defect (diabetes?) was injected with million or billions of gene fragments in the hopes that one would imbed itself within the DNA of a cell and cause that cell to begin to produce the proper protein (is this called transformation?).

Has gene therapy gotten any further in 20 years? I haven’t heard of any great breakthroughs in the news. Have they completely abandoned this transformation idea? What about using viruses to insert genes? What’s the latest in this area?

2

There are a lot of people still working in gene therapy, and I believe some diseases have actually been cured. There were some setbacks though because some groups were using adenovirus as a delivery system and they gave someone way too much and he died. I worked on a project a few years ago that was using adeno-associated virus (safe) as a delivery system, it is large enough to hold the amount of DNA that would be required.

There’s a great review here

and here is a story of a boy being cured by gene therapy.