Featured Research

from universities, journals, and other organizations

Persistent gene therapy in muscle may not require immunosuppression

Date:
November 15, 2013
Source:
Journal of Clinical Investigation
Summary:
In a new study, researchers evaluated the persistence of rAAV-mediated expression the gene encoding M-type ±-1 antitrypsin in patients that were AAT deficient

Successful gene therapy is based on the effective delivery and maintained expression of healthy copies of a gene into tissues of individuals with a disease-associated genetic mutation. Recombinant adeno-associated virus (rAAV) vectors have shown promise in early clinical trials as effective therapies for several genetic diseases, including Leber congenital amaurosis, Parkinson disease, and hemophilia. Unfortunately, delivery of rAAV vectors to tissues other than the retina and CNS often results in development of an immune response against the viral capsid. The development of a neutralizing response against the rAAV vector prevents sustained expression of the healthy gene in the absence of immunosuppression.

Related Articles


In this issue of the Journal of Clinical Investigation, Christian Mueller and colleagues at the University of Massachusetts Medical School evaluated the persistence of rAAV-mediated expression the gene encoding M-type α-1 antitrypsin (M-AAT) in patients that were AAT deficient. Patients received multiple intramuscular doses without immunosuppression, and M-ATT expression was evaluated in muscle biopsies. The authors determined that subjects sustained M-ATT expression in muscle tissue for at least one year, despite an initial influx of immune cells. Further evaluation of muscle fibers revealed a substantial population of regulatory T cells in patients with persistent M-ATT expression.

Together, the results from this study suggests that delivery of an M-ATT-encoding rAAV vector promotes a regulatory immune response that allows for long term gene expression that does not require immune suppression.


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 Reference:

  1. Christian Mueller, Jeffrey D. Chulay, Bruce C. Trapnell, Margaret Humphries, Brenna Carey, Robert A. Sandhaus, Noel G. McElvaney, Louis Messina, Qiushi Tang, Farshid N. Rouhani, Martha Campbell-Thompson, Ann Dongtao Fu, Anthony Yachnis, David R. Knop, Guo-jie Ye, Mark Brantly, Roberto Calcedo, Suryanarayan Somanathan, Lee P. Richman, Robert H. Vonderheide, Maigan A. Hulme, Todd M. Brusko, James M. Wilson, Terence R. Flotte. Human Treg responses allow sustained recombinant adeno-associated virus–mediated transgene expression. Journal of Clinical Investigation, 2013; DOI: 10.1172/JCI70314

Cite This Page:

Journal of Clinical Investigation. "Persistent gene therapy in muscle may not require immunosuppression." ScienceDaily. ScienceDaily, 15 November 2013. <www.sciencedaily.com/releases/2013/11/131115130201.htm>.
Journal of Clinical Investigation. (2013, November 15). Persistent gene therapy in muscle may not require immunosuppression. ScienceDaily. Retrieved November 26, 2014 from www.sciencedaily.com/releases/2013/11/131115130201.htm
Journal of Clinical Investigation. "Persistent gene therapy in muscle may not require immunosuppression." ScienceDaily. www.sciencedaily.com/releases/2013/11/131115130201.htm (accessed November 26, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Wednesday, November 26, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

From Popcorn To Vending Snacks: FDA Ups Calorie Count Rules

From Popcorn To Vending Snacks: FDA Ups Calorie Count Rules

Newsy (Nov. 25, 2014) — The US FDA is announcing new calorie rules on Tuesday that will require everywhere from theaters to vending machines to include calorie counts. Video provided by Newsy
Powered by NewsLook.com
Daily Serving Of Yogurt Could Reduce Risk Of Type 2 Diabetes

Daily Serving Of Yogurt Could Reduce Risk Of Type 2 Diabetes

Newsy (Nov. 25, 2014) — Need another reason to eat yogurt every day? Researchers now say it could reduce a person's risk of developing type 2 diabetes. Video provided by Newsy
Powered by NewsLook.com
Madagascar Working to Contain Plague Outbreak

Madagascar Working to Contain Plague Outbreak

AFP (Nov. 24, 2014) — Madagascar said Monday it is trying to contain an outbreak of plague -- similar to the Black Death that swept Medieval Europe -- that has killed 40 people and is spreading to the capital Antananarivo. Duration: 00:42 Video provided by AFP
Powered by NewsLook.com
Are Female Bosses More Likely To Be Depressed?

Are Female Bosses More Likely To Be Depressed?

Newsy (Nov. 24, 2014) — A new study links greater authority with increased depressive symptoms among women in the workplace. Video provided by Newsy
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