# What's new in gene therapy?

**URL:** <https://boards.straightdope.com/t/whats-new-in-gene-therapy/356153>\
**Category:** Factual Questions\
**Created:** [May 10, 2006, 1:06am UTC](https://boards.straightdope.com/t/whats-new-in-gene-therapy/356153 "2006-05-10T01:06:33Z")\
**Posts on this page:** 2\
**Page:** 1

<div class="post-metadata">

**Author:** ![solkoe](https://avatars.discourse-cdn.com/v4/letter/s/919ad9/32.png) [@solkoe](https://boards.straightdope.com/u/solkoe)\
**Post date:** [May 10, 2006, 1:06am UTC](https://boards.straightdope.com/t/whats-new-in-gene-therapy/356153/1 "2006-05-10T01:06:33Z")

</div>

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?

---

<div class="post-metadata">

**Author:** ![Bob55](https://avatars.discourse-cdn.com/v4/letter/b/e9c0ed/32.png) [@Bob55](https://boards.straightdope.com/u/Bob55)\
**Post date:** [May 10, 2006, 7:39am UTC](https://boards.straightdope.com/t/whats-new-in-gene-therapy/356153/2 "2006-05-10T07:39:16Z")

</div>

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](http://en.wikipedia.org/wiki/Gene_therapy)

and [here is a story](http://news.bbc.co.uk/1/hi/health/4101411.stm) of a boy being cured by gene therapy.
