Featured Research

from universities, journals, and other organizations

New Technique Adds Precision And Permanence To Gene Therapy

Date:
October 11, 2005
Source:
The Mount Sinai Hospital / Mount Sinai School of Medicine
Summary:
Mount Sinai School of Medicine researchers developed a technique for inserting genes into specific sites on the genome in liver cells. The genes are inserted into non-coding regions of the genome so there is no danger of interfering with the functioning of other genes. Once inserted, the gene remains a permanent part of the cell's genome. In a study published in this week's PNAS, the researchers used this technique to cure phenylketonuria (PKU) in mice.

Mount Sinai School of Medicine researchers developed a technique forinserting genes into specific sites on the genome in liver cells. Thegenes are inserted into non-coding regions of the genome so there is nodanger of interfering with the functioning of other genes. Onceinserted, the gene remains a permanent part of the cell's genome. In astudy published in this week's PNAS, the researchers used thistechnique to cure phenylketonuria (PKU) in mice.

Related Articles


"To date gene therapy has relied upon vectors that randomly insertgenes into the cell's genome," explains Savio L. C. Woo, PhD, Professorand Chairman of Gene and Cell Medicine at Mount Sinai School ofMedicine and corresponding author on the study. "The technique wedeveloped identifies a specific sequence which only occurs in a fewplaces in the mammalian genome. These sequences occur between genes sothere is no danger of the insertion of the gene damaging existing genesin the cell.

"Because the genes are inserted permanently, a few applications wouldsuffice to permanently correct a disease." Dr. Woo and his colleague LiChen , PhD, a post-doctoral fellow at Mount Sinai) were able to curePKU in mice with just three intravenous injections. The levels ofphenylalanine in the treated mice dropped to normal range and remainedstable thereafter. Their fur color also changed from gray to black,indicating that they were now producing normal levels of melanin, apigmentation which is under-produced in mice and humans with PKU.

Drs. Woo and Chen used a gene from a bacteriophage that recognizes aspecific DNA sequence. This sequence occurs only several times in theentire mouse genome and it is always found in the non-coding regionbetween genes. Similar sequences are found in a few locations in thehuman genome that are also between existing genes.

"The current challenge is to identify a suitable means of introducingDNA into liver cells," said Dr. Woo. "Once that technology isdeveloped, this new technique will provide a safe and efficient meansof integrating the DNA into the cell's genome."

In addition to PKU, this technique could be used to cure other geneticdiseases caused by missing liver enzymes including hemophilia and ureacycle enzyme deficiencies, as well as cholesterol clearing from theblood and others.


Story Source:

The above story is based on materials provided by The Mount Sinai Hospital / Mount Sinai School of Medicine. Note: Materials may be edited for content and length.


Cite This Page:

The Mount Sinai Hospital / Mount Sinai School of Medicine. "New Technique Adds Precision And Permanence To Gene Therapy." ScienceDaily. ScienceDaily, 11 October 2005. <www.sciencedaily.com/releases/2005/10/051011001005.htm>.
The Mount Sinai Hospital / Mount Sinai School of Medicine. (2005, October 11). New Technique Adds Precision And Permanence To Gene Therapy. ScienceDaily. Retrieved January 31, 2015 from www.sciencedaily.com/releases/2005/10/051011001005.htm
The Mount Sinai Hospital / Mount Sinai School of Medicine. "New Technique Adds Precision And Permanence To Gene Therapy." ScienceDaily. www.sciencedaily.com/releases/2005/10/051011001005.htm (accessed January 31, 2015).

Share This


More From ScienceDaily



More Health & Medicine News

Saturday, January 31, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

CDC: Get Vaccinated for Measles

CDC: Get Vaccinated for Measles

Reuters - US Online Video (Jan. 30, 2015) The CDC is urging people to get vaccinated for measles amid an outbreak that began at Disneyland and has now infected more than 90 people. Linda So reports. Video provided by Reuters
Powered by NewsLook.com
Obama To Outline New Plan For Personalized Medicine

Obama To Outline New Plan For Personalized Medicine

Newsy (Jan. 30, 2015) President Obama is expected to speak with drugmakers Friday about his Precision Medicine Initiative first introduced last week. Video provided by Newsy
Powered by NewsLook.com
NFL Concussions Down; Still on Parents' Minds

NFL Concussions Down; Still on Parents' Minds

AP (Jan. 30, 2015) The NFL announced this week that the number of game concussions dropped by a quarter over last season. Still, the dangers of the sport still weigh on players, and parents&apos; minds. (Jan. 30) Video provided by AP
Powered by NewsLook.com
U.S. Wants to Analyze DNA from 1 Million People

U.S. Wants to Analyze DNA from 1 Million People

Reuters - US Online Video (Jan. 30, 2015) The U.S. has proposed analyzing genetic information from more than 1 million American volunteers to learn how genetic variants affect health and disease. Rough Cut (no reporter narration). Video provided by Reuters
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