“Gene Therapy in a New Light”

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The Story

“Gene Therapy in a New Light”
by Jocelyn Kaiser
Smithsonian, January 2009

The Pitch

[Kaiser notes that this pitch was unusually informal because she and Smithsonian editor Laura Helmuth are friends.]

Hi Laura,

Would Smithsonian mag want a story on gene therapy? It may be a long shot, but I thought I’d throw it out there. This week I and others wrote about two small studies in the NEJM reporting that gene therapy partially restored the sight of four nearly blind patients http://sciencenow.sciencemag.org/cgi/content/full/2008/428/2. The studies were in the UK and U Penn. I visited the Penn leaders for a possible profile (they are a very engaging husband/wife) but instead ended up writing a print story about some politics behind the study. But I’m thinking about pitching a profile somewhere else.

The story would also describe this couple’s earlier work in (cute, charismatic) DOGS (they restored sight in more than 50 with the same blindness disease). They are treating younger patients this month, so they may have more dramatic results in a few weeks (but I realize Smithsonian’s deadlines mean that any news would be old already).

Gene therapy is not as sexy as stem cells right now, but in the past few years it has chalked up its first successes, including curing about 20 kids with bubble boy disease (but it also caused leukemia in four). Treating blindness could be the next success story.

So you know, the blindness studies have gotten a lot of press in the UK and US–ABC has covered it, and I know of at least one profile: http://www.philly.com/inquirer/breaking/news_breaking/20080426_Phila researchers_bring_ sight_to_the_blind.html

One question if I wrote about it for you–would you want me to be there to watch a patient before and after treatment? It would be good to include but would be a fairly big time commitment for me (two trips to Philly).

Let me know what you think (or have thoughts on where else I could pitch this! Discover?)

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