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:5/16/2017)... TEANECK, N.J. , May 16, 2017  Veratad ... leading provider of online age and identity verification solutions, ... the K(NO)W Identity Conference 2017, May 15 thru May ... Ronald Regan Building and International Trade Center. ... across the globe and in today,s quickly evolving digital ...
(Date:5/6/2017)... , May 5, 2017 RAM ... announced a new breakthrough in biometric authentication based ... quantum mechanical properties to perform biometric authentication. These new ... semiconductor material created by Ram Group and its ... entertainment, transportation, supply chains and security. Ram Group ...
(Date:4/19/2017)... 2017 The global military biometrics ... marked by the presence of several large global players. ... five major players - 3M Cogent, NEC Corporation, M2SYS ... nearly 61% of the global military biometric market in ... global military biometrics market boast global presence, which has ...
Breaking Biology News(10 mins):
(Date:10/11/2017)... ... 11, 2017 , ... A new study published in Fertility ... fresh in vitro fertilization (IVF) transfer cycles. The multi-center matched cohort ... After comparing the results from the fresh and frozen transfer cohorts, the authors ...
(Date:10/10/2017)... ... October 10, 2017 , ... ... company advancing targeted antibody-drug conjugate (ADC) therapeutics, today confirmed licensing rights that ... Polymerized Liposomal Nanoparticle), a technology developed in collaboration with Children’s Hospital Los ...
(Date:10/10/2017)... 10, 2017 International research firm Parks Associates announced ... at the TMA 2017 Annual Meeting , October 11 in ... residential home security market and how smart safety and security products impact ... Parks Associates: Smart Home ... "The residential security market has ...
(Date:10/10/2017)... PA (PRWEB) , ... October 10, 2017 , ... ... year’s recipients of 13 prestigious awards honoring scientists who have ... presented in a scheduled symposium during Pittcon 2018, the world’s leading conference and ...
Breaking Biology Technology:
Cached News: