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:
TAG: New technique adds precision and permanence gene therapy

(Date:4/17/2014)... State University engineer has developed a patented technique ... explosive devices. The same technique could help police ... M. and Kay L. Theede chair in engineering ... and his research team have created a template-based ... car trunks. The distance detection method called ...
(Date:4/17/2014)... in Europe, Asia and the United States are spreading ... impacting native honeybee populations at this time, according to ... including Nosema microsporidia and Varroa ... to these invasive pests, which suggests to us that ... and the United States currently are not necessary in ...
(Date:4/17/2014)... reporting in the Cell Press journal Current Biology ... with rather novel sex lives. The Brazilian insects, which ... Neotrogla , are the first example of an ... has been identified in several different animals, Neotrogla ... organ is also reversed," says Kazunori Yoshizawa from Hokkaido ...
Breaking Biology News(10 mins):Patented research remotely detects nitrogen-rich explosives 2East African honeybees are safe from invasive pests… for now 2East African honeybees are safe from invasive pests… for now 3In sex-reversed cave insects, females have the penises 2
(Date:1/15/2014)... (PRWEB) January 15, 2014 This webinar ... nonclinical and clinical safety assessment in biosimilars. , Regulatory ... for biosimilar drug development, however the complex nature of ... quality, safety and efficacy extremely challenging. Based on the ...
(Date:1/15/2014)... DTS Language Services, Inc . is pleased ... Life Science organizations who need document translations. Clients will ... their documents in advance with a selection of nearly 50 ... often a critical factor in clinical and scientific fields, and ...
(Date:1/14/2014)... January 14, 2014 Date: Friday, April 11, ... Warrington Country Club, 1360 Almshouse Road, Warrington, Pa. ... organization solely dedicated to finding a cure for hepatitis B ... will host its annual Crystal Ball on Friday, April 11 ...
(Date:1/14/2014)... New York, NY (PRWEB) January 14, 2014 ... coverage of Alliqua, Inc. (OTCQB: ALQA). Alliqua is an ... products to serve the wound care market. , Free ... Alliqua was restructured with a seasoned management team and ...
Breaking Biology Technology:Xtalks Life Sciences Webinar Examines Safety Assessment of Biosimilars 2DTS Improves Efficiency for Life Science Document Translations 2Hepatitis B Foundation to Host Annual Crystal Ball Gala 2EquitiesIQ Initiates Coverage of Alliqua, Inc. 2EquitiesIQ Initiates Coverage of Alliqua, Inc. 3
Cached News: