Featured Research

from universities, journals, and other organizations

Making Gene Therapy Safer Using Self-inactivating LTRs

Date:
April 2, 2009
Source:
Journal of Clinical Investigation
Summary:
Several patients in gene therapy clinical trials have developed leukemia as a result of their treatment. The underlying cause of leukemia is thought to be that the viral vectors used to carry the therapeutic gene into cells (gamma-RVs) integrate into the genome of the cells disrupting the natural control of cancer-associated genes (a process known as insertional mutagenesis).

Several patients in gene therapy clinical trials have developed leukemia as a result of their treatment. The underlying cause of leukemia is thought to be that the viral vectors used to carry the therapeutic gene into cells (gamma-RVs) integrate into the genome of the cells disrupting the natural control of cancer-associated genes (a process known as insertional mutagenesis).

By analyzing specific elements of gamma-RVs and another type of viral vector, LVs, in a tumor-prone mouse model, a team of researchers, at San Raffaele-Telethon Institute for Gene Therapy, Italy, has now provided evidence that LVs are substantially less likely to cause insertional mutagenesis and tumors than gamma-RVs.

Further, they found that modifying an element (known as the LTR) of both LVs and gamma-RVs such that it is self-inactivating (SIN) further improved safety. The authors therefore conclude that SIN viral vectors should be the preferred choice in future gene therapy trials.

In an accompanying commentary, Ute Modlich and Christopher Baum, at Hannover Medical School, Germany, discuss the clinical importance of these data and the numerous questions that they pose for future research.

The research is published in the March 23, 2009, issue of the Journal of Clinical Investigation.


Story Source:

The above story is based on materials provided by Journal of Clinical Investigation. Note: Materials may be edited for content and length.


Journal References:

  1. Montini et al. The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. Journal of Clinical Investigation, 2009; DOI: 10.1172/JCI37630
  2. Ute Modlich, Christopher Baum. Preventing and exploiting the oncogenic potential of integrating gene vectors. Journal of Clinical Investigation, 2009; DOI: 10.1172/JCI38831

Cite This Page:

Journal of Clinical Investigation. "Making Gene Therapy Safer Using Self-inactivating LTRs." ScienceDaily. ScienceDaily, 2 April 2009. <www.sciencedaily.com/releases/2009/03/090324213208.htm>.
Journal of Clinical Investigation. (2009, April 2). Making Gene Therapy Safer Using Self-inactivating LTRs. ScienceDaily. Retrieved October 1, 2014 from www.sciencedaily.com/releases/2009/03/090324213208.htm
Journal of Clinical Investigation. "Making Gene Therapy Safer Using Self-inactivating LTRs." ScienceDaily. www.sciencedaily.com/releases/2009/03/090324213208.htm (accessed October 1, 2014).

Share This



More Health & Medicine News

Wednesday, October 1, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Pregnancy Spacing Could Have Big Impact On Autism Risks

Pregnancy Spacing Could Have Big Impact On Autism Risks

Newsy (Oct. 1, 2014) A new study says children born less than one year and more than five years after a sibling can have an increased risk for autism. Video provided by Newsy
Powered by NewsLook.com
Robotic Hair Restoration

Robotic Hair Restoration

Ivanhoe (Oct. 1, 2014) A new robotic procedure is changing the way we transplant hair. The ARTAS robot leaves no linear scarring and provides more natural results. Video provided by Ivanhoe
Powered by NewsLook.com
Insertable Cardiac Monitor

Insertable Cardiac Monitor

Ivanhoe (Oct. 1, 2014) A heart monitor the size of a paperclip that can save your life. The “Reveal Linq” allows a doctor to monitor patients with A-Fib on a continuous basis for up to 3 years! Video provided by Ivanhoe
Powered by NewsLook.com
Attacking Superbugs

Attacking Superbugs

Ivanhoe (Oct. 1, 2014) Two weapons hospitals can use to attack superbugs. Scientists in Ireland created a new gel resistant to superbugs, and a robot that can disinfect a room in minutes. Video provided by Ivanhoe
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins