Entering An Era in Gene Therapy; Bringing gene therapy in from the cold: a BBC science report via humanrescueintermedia


13 October 2011 Last updated at 09:09 GMT Tom FeildenArticle written byTom FeildenScience correspondent, Today programme

Bringing gene therapy in from the cold


Not so long ago gene therapy was the "Next Big Thing" in medical science. Revolutionary new treatments and cures, we were told, were just around the corner.

But when the 18-year-old Jesse Gelsinger died during a clinical trial for a revolutionary new gene therapy technique in 1999, that bubble of expectation burst. The mantle of medical hyperbole – and the funding – moved on to other, as yet untarnished advances, including stem cells and the promise of personalised medicine.

Although investment in gene therapy withered, away from the limelight research has continued and in the intervening years a number of important breakthroughs and treatments have been developed. The latest of these, described in a paper published in the journal Nature, may go some way towards reinstating gene therapy in medical science’s premier league.

Scientists at the Wellcome Trust’s Sanger Institute and the University of Cambridge have succeeded in correcting the genetic mutation responsible for Antitrypsin deficiency – a debilitating condition that causes cirrhotic liver disease and lung emphysema in some 30,000 patients in the UK alone.

Starting with a skin biopsy from a patient suffering from the disease the team derived a population of iPS, or induced pluripotent stem cells, containing faulty copies of the alpha-1 antitrypsin gene.

Using molecular scissors they then removed the abnormal copies of the gene and replaced them with corrected versions, before stimulating the stem cells to differentiate into liver cells.

Finally they implanted these healthy liver cells into a mouse model – demonstrating not only that they functioned properly, but that over time, they re-populated the entire organ generating a disease-free liver.

Speaking on the programme this morning Cambridge University’s Professor David Lomas hailed the result as a remarkable breakthrough.

"The genetic correction is really quite a phenomenal technical advance. Now we have to understand how we can safely use these cells in patients, because it’s most important we don’t do harm. The cells that we put back in have to be effective, have to function well, but most of all have to cause no harm to the patient."

It’s early days, but if researchers can overcome the technical obstacles and safety concerns, the technique could offer patients a viable alternative to a liver transplant and a lifetime on immuno-suppressant drugs – and help bring gene therapy in from the cold.

Your comments
Tom FeildenArticle written by Tom FeildenTom FeildenScience correspondent, Today programme

Unravelling the epigenome

12:34 UK time, Wednesday, 12 October 2011

Undertanding the genetic code’s chemical instruction manual

Read full article


Be the first to comment

Sign in or register to comment and rate comments.

Go to link for the authentic view of article


The Human Rescue Team
A Self Styled Laboratory of Neuroinformatics- Interlecting in Nano Age World


We honored your feedback!

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out /  Change )

Google+ photo

You are commenting using your Google+ account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )


Connecting to %s

%d bloggers like this: