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:1/22/2015)... , Jan. 21, 2015  Analyst Report Issued by Small ... Brands report showed that planet-wide, transactions at merchants on the ... the total number of credit, debit, and prepaid cards reached ... were victims of fraudulent card usage in 2012, and credit ...
(Date:1/22/2015)... Infinisource has launched its new NXG series of time clocks ... a higher standard for collecting attendance and labor data and ... installation, touch screen interface and seamless connection to cloud-based iSolved, ... solution for the small to mid-size employer. The ...
(Date:12/22/2014)... DENVER , Dec. 22, 2014  The 2014 Holiday ... industry as Acuity Market Intelligence reports that the long ... Acuity forecasts that intensifying demand for smart phones, tablets, ... a global market of 2.5 billion users with nearly ...
Breaking Biology News(10 mins):$4.80 Near Term Price Target with "Buy" given by CapRock Research for NXT-ID, Inc. (NASDAQ: NXTD) issued by Small Cap IR 2Infinisource's NXG series sets new time clock standard 2First Season of Holiday Shopping with Mobile Biometric Payments Wraps Up With a Present for Biometrics Industry: A Rosy Forecast for More Than 2 Billion Users of 4.8 Billion Mobile Biometric Devices by 2020 2
(Date:1/22/2015)... BRUNSWICK , Nueva Jersey , 22 de ... Award for Biomedical Research abre hoy su llamada a ... personas cuya investigación científica ha hecho, o tiene el ... la salud humana. Las nominaciones se aceptarán hasta el ...
(Date:1/22/2015)...   GenoSpace , a precision medicine software company that has ... use of genomic, imaging and other biomedical data in research ... Munson , CEO of Aspera, an IBM Company, to its ... http://photos.prnewswire.com/prnh/20150122/170713 "We are pleased to welcome ...
(Date:12/24/2014)...  United Therapeutics Corporation (NASDAQ: UTHR ) announced ... has submitted a pre-market approval application to the U.S. ... Medtronic,s SynchroMed ® II implantable drug infusion system ... Therapeutics, Remodulin ® (treprostinil) Injection delivered intravenously to ...
(Date:12/24/2014)... 24, 2014   BioLife Solutions , Inc. (NASDAQ: ... clinical grade hypothermic storage and cryopreservation freeze ... cells and tissues  ("BioLife" or the "Company"), today announced that ... May 4, 2015 (the "Annual Meeting"). Because the ...
Breaking Biology Technology:El Dr. Paul Janssen Award for Biomedical Research emite su llamada a los nominados para 2015 2El Dr. Paul Janssen Award for Biomedical Research emite su llamada a los nominados para 2015 3El Dr. Paul Janssen Award for Biomedical Research emite su llamada a los nominados para 2015 4GenoSpace Expands Board with Appointment of Michelle Munson 2United Therapeutics Announces Submission Of Pre-Market Approval Application For Implantable Drug Infusion System To Deliver Remodulin 2United Therapeutics Announces Submission Of Pre-Market Approval Application For Implantable Drug Infusion System To Deliver Remodulin 3United Therapeutics Announces Submission Of Pre-Market Approval Application For Implantable Drug Infusion System To Deliver Remodulin 4BioLife Solutions Sets Date for Annual Meeting of Stockholders 2
Cached News: