PLEDGE NOW
Here and Now with Robin Young and Jeremy Hobson
Public radio's live
midday news program
With sponsorship from
Mathworks - Accelerating the pace of engineering and science
Accelerating the pace
of engineering and science
Wednesday, July 17, 2013

New Genetic Therapy Could Erase Down Syndrome

Erin Witkowski, of Port Jervis, N.Y., and her 16-month-old son Grady pose for photos in New York's Central Park, June 6, 2011. (Richard Drew/AP)

Erin Witkowski, of Port Jervis, N.Y., and her 16-month-old son Grady pose for photos in New York’s Central Park, June 6, 2011. (Richard Drew/AP)

Down syndrome affects nearly six million people worldwide. The genetic disorder impairs physical growth and intelligence, and it can cause serious medical problems, from heart defects to early dementia.

Down syndrome is caused by a chromosome defect and today, the journal Nature reports that researchers may have found a way to correct that defect, though it will be years before it could be used as a therapy in humans.

It’s an application of the idea behind gene therapy. The premise is that if you have a disease that’s caused by a defect in a single gene, a virus engineered to carry a “fix-it” gene can be sent in to remove and replace the abnormal gene.

The new application of this idea is called chromosome therapy.

Down syndrome is caused by the presence of a third copy of chromosome number 21. Chromosome therapy could be used to “silence” or turn off the extra copy, which contains hundreds of genes, with the hope that that would turn off the effects of Down Syndrome.

Guest


Please follow our community rules when engaging in comment discussion on this site.
Robin and Jeremy

Robin Young and Jeremy Hobson host Here & Now, a live two-hour production of NPR and WBUR Boston.

May 27 Comment

How Trump, Clinton And Sanders Change Their Voices To Win Over Voters

Charisma is a crucial component of a politician's appeal to voters. But there's more than one way to inspire confidence.

May 27 52 Comments

New York Considers A Ban On Declawing Cats

Some pet owners have routinely had their cats declawed. But opponents say the procedure isn't so simple.

May 27 3 Comments

New Drug-Resistant Bacteria Seen In U.S. For The First Time

The CDC's director has expressed concern, saying it shows that we're close to the "end of the road" for antibiotics.

May 26 6 Comments

As Lethal Heroin Overdose Numbers Rise, Families Find Solace In Organ Donation

Organ banks around the country have noted an increasing number of organs from donors who have died of overdoses.