HOME >> BIOLOGY >> NEWS
New technique adds precision and permanence to gene therapy

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.

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

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

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

"The current challenge is to identify a suitable means of introducing DNA into liver cells," said Dr. Woo. "Once that technology is devel
'"/>

Contact: Mount Sinai Press Office
newsmedia@mssm.edu
212-241-9200
The Mount Sinai Hospital / Mount Sinai School of Medicine
10-Oct-2005


Page: 1 2

Related biology news :

1. Innovative tagging technique may help researchers better protect fish stocks
2. Innovative research technique reveals another natural wonder in Yellowstone Park
3. New techniques redefine assessment of liver disease
4. New technique will produce a better chromosome map
5. Researchers develop technique for bacteria crowd control
6. Bioluminescence at the service of a novel cerebral imaging technique
7. Natural antibiotics yield secrets to atom-level imaging technique
8. New imaging technique tracks traffic patterns of white blood cells
9. Vet College gets grant to develop fish virus diagnostic technique
10. Harvard team creates spray drying technique for TB vaccine
11. Novel laboratory technique nudges genes into activity

Post Your Comments:
*Name:
*Comment:
*Email:


(Date:8/23/2017)... Aug. 23, 2017  The general public,s help is being enlisted in ... that live in and on the human body –and are believed to ... The Microbiome Immunity Project ... microbiome, starting with the gut. The project's goal is to help advance ... IBM ...
(Date:6/14/2017)... 15, 2017  IBM (NYSE: IBM ) is introducing several ... dedicated to developing collaboration between startups and global businesses, taking ... During the event, nine startups will showcase the solutions they ... industries. France is ... with a 30 percent increase in the number of startups ...
(Date:4/24/2017)... 24, 2017 Janice Kephart , ... Identity Strategy Partners, LLP (IdSP) , today issues ... President Trump,s March 6, 2017 Executive Order: ... vetting can be instilled with greater confidence, enabling ... all refugee applications are suspended by until at ...
Breaking Biology News(10 mins):
(Date:10/10/2017)... research firm Parks Associates announced today that Tom Kerber ... Annual Meeting , October 11 in Scottsdale, Arizona . ... how smart safety and security products impact the competitive landscape. ... Parks Associates: Smart Home Devices: Main Purchase Driver ... "The residential security market has experienced continued growth, and the ...
(Date:10/10/2017)... ... October 10, 2017 , ... The ... prestigious awards honoring scientists who have made outstanding contributions to analytical ... during Pittcon 2018, the world’s leading conference and exposition for laboratory science, which ...
(Date:10/9/2017)... ... October 09, 2017 , ... At its national board meeting in North Carolina, ... Harvard University’s Departments of Physics and Astronomy, has been selected for membership in ... team for the 2015 Breakthrough Prize in Fundamental physics for the discovery of the ...
(Date:10/7/2017)... ... October 06, 2017 , ... ... advanced instruments and applications consulting for microscopy and surface analysis, Nanoscience Instruments ... consulting, Nanoscience Analytical offers a broad range of contract analysis services for ...
Breaking Biology Technology:
Cached News: