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
The Mount Sinai Hospital / Mount Sinai School of Medicine

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:

(Date:3/29/2016)... 2016 LegacyXChange, Inc. (OTC: ... SelectaDNA/CSI Protect are pleased to announce our successful effort ... variety of writing instruments, ensuring athletes signatures against counterfeiting ... from athletes on LegacyXChange will be assured of ongoing ... Bill Bollander , CEO states, "By ...
(Date:3/22/2016)... PUNE, India , March 22, 2016 ... new market research report "Electronic Sensors Market for ... Fingerprint, Proximity, & Others), Application (Communication & ... and Geography - Global Forecast to 2022", ... consumer industry is expected to reach USD ...
(Date:3/21/2016)... , March 22, 2016 ... recognition with passcodes for superior security   ... a leading provider of secure digital communications services, today ... biometric technology and offer enterprise customers, particularly those in ... facial recognition and voice authentication within a mobile app, ...
Breaking Biology News(10 mins):
(Date:6/27/2016)... ... June 27, 2016 , ... ... medical technologies, services and solutions to the healthcare market. The company's primary focus ... distribution, manufacturing, sales and marketing strategies that are necessary to help companies efficiently ...
(Date:6/24/2016)... DIEGO , June 24, 2016 ... more sensitively detects cancers susceptible to PARP inhibitors ... circulating tumor cells (CTCs). The new test has ... HRD-targeted therapeutics in multiple cancer types. ... targeting DNA damage response pathways, including PARP, ATM, ...
(Date:6/23/2016)... 2016   Boston Biomedical , an industry ... to target cancer stemness pathways, announced that its ... Drug Designation from the U.S. Food and Drug ... including gastroesophageal junction (GEJ) cancer. Napabucasin is an ... cancer stemness pathways by targeting STAT3, and is ...
(Date:6/23/2016)... Prostate Cancer Foundation (PCF) is pleased to announce 24 new Young Investigator ... Members of the Class of 2016 were selected from a pool of 128 ... About the Class of 2016 PCF Young Investigators ... ... ...
Breaking Biology Technology:
Cached News: