The Latest Biology News And Medical NewsBiology News 2Health News 2Biology News 3Health News 3
HOME >> BIOLOGY >> NEWS
Gene Therapy Animal Study Offers Hope For Some Neurodegenerative Diseases

A study co-authored by UC San Francisco researchers offers the first evidence that a gene therapy technique involving ribozymes, molecules that disrupt protein production, can be used to slow neurodegeneration in an animal model, signalling a possible approach for overcoming one of the monumental hurdles of gene therapy.

In the study, reported in the August issue of Nature Medicine, researchers demonstrated that ribozymes could be used to reduce the production of a faulty gene product that leads to the degeneration of light-sensitive rod cells in the eye. The degeneration occurs in association with autosomal dominant retinitis pigmentosa, an untreatable condition of progressive blindness.

Importantly, the approach could potentially also be used to treat other diseases resulting from a destructive protein, including some forms of glaucoma, Huntington's disease, amyotrophic lateral sclerosis (ALS) and Warfan's syndrome, according to the senior author of the study, Matthew M. LaVail, PhD, a professor of anatomy and ophthalmology at UCSF.

The finding represents an important breakthrough in the still fledgling field of gene therapy, which has been particularly challenged by disorders caused by abnormal, or mutant, proteins, so-called autosomal dominant diseases, said LaVail.

Most gene therapy approaches have focused on diseases that occur when the two copies of a particular gene fail to produce, or fail to produce sufficient quantity of, a healthy protein. The challenge in developing treatments for these so-called autosomal recessive diseases, such as cystic fibrosis, has involved trying to introduce healthy genes directly into the pertinent cells to replace and compensate for the missing proteins. This challenge has been daunting enough. Some successes have been achieved in animal models with recessive diseases, but only in recent months have such approaches even begun to demonstrate small successes in clinical trials, according to LaVail.

But res
'"/>

Contact: Jennifer O'Brien
jobrien@itsa.ucsf.edu
(415) 476-2557
University of California - San Francisco
15-Sep-1998


Page: 1 2 3

Related biology news :

1. 1st International Conference on Cell Therapy for Cardiovascular Disease
2. National Cancer Gene Therapy Foundation gives $4.0 million in research grants
3. Braunschweig Prize 2003 for Molecular Cancer Therapy
4. Alliance for Cancer Gene Therapy announces $1.5 million in national grants to young investigators
5. New research on safety of intracoronary delivery of adenovector reported in cover story of Molecular Therapy
6. Therapy hinders AIDS virus from evolving drug resistance, Hopkins researchers find
7. Invitation To Cover: American Society Of Gene Therapy Meeting
8. Gene Therapy For Pain
9. Role Of Protein Linked To Colon Cancer Identified, Offering Potential Target For Therapy
10. Shock Therapy Exceeds Expectations In Cleaning Up Contaminated Soils
11. Penn Researchers Develop Gene Therapy Technique That Reverses Muscle Membrane Weakness In Muscular Dystrophy Variant

Post Your Comments:
(Date:11/22/2009)..., Census of Marine Life scientists have inventori...ution of deep sea species that have never known su... a frigid black world down to 5,000 meters (~3 mil...towed cameras, sonar and other vanguard technologi...arkness now number 17,650, a diverse collection of...
(Date:11/21/2009)...artinsried, Germany, November 20,2009 / b3c newsw...e completion of a financing round, gaining two new...st. At the same time KINAXO reports that Jrgen Sch...mpanys advisory board. , KINAXO shareholders Axe...l as the companys institutional investors Mountain...
(Date:11/20/2009)... A decade ago, a group of University of Oklahoma r...ome as part of the human genome project. Today, ...ntributing to an international effort to sequence ...d by the National Science Foundation for plant gen... importance, so improving the tomato and crop yiel...
Breaking Biology News(10 mins):Beyond sunlight: Explorers census 17,650 ocean species between edge of darkness and black abyss 2Beyond sunlight: Explorers census 17,650 ocean species between edge of darkness and black abyss 3Beyond sunlight: Explorers census 17,650 ocean species between edge of darkness and black abyss 4Beyond sunlight: Explorers census 17,650 ocean species between edge of darkness and black abyss 5Beyond sunlight: Explorers census 17,650 ocean species between edge of darkness and black abyss 6Beyond sunlight: Explorers census 17,650 ocean species between edge of darkness and black abyss 7Beyond sunlight: Explorers census 17,650 ocean species between edge of darkness and black abyss 8Beyond sunlight: Explorers census 17,650 ocean species between edge of darkness and black abyss 9KINAXO Completes Financing Round to Enhance Biomarker Development 2KINAXO Completes Financing Round to Enhance Biomarker Development 3NSF awards $7.5M grant to University of Oklahoma for plant genomics 2Breeding their horns off a winner 9614 1Breeding their horns off a winner 9614 2New Omega 3 Blood Test 3A A Better Predictor of Coronary Heart Disease Than Cholesterol 54853 1New Omega 3 Blood Test 3A A Better Predictor of Coronary Heart Disease Than Cholesterol 54853 2New Omega 3 Blood Test 3A A Better Predictor of Coronary Heart Disease Than Cholesterol 54853 3New Omega 3 Blood Test 3A A Better Predictor of Coronary Heart Disease Than Cholesterol 54853 4New Jersey Surgery Centers Pick Health Informatics Consulting to Implement Patient Electronic Health Record Systems 13630 1New Jersey Surgery Centers Pick Health Informatics Consulting to Implement Patient Electronic Health Record Systems 13630 2
(Date:11/19/2009)... BETHESDA, Md., Nov. 19 ...r in biotechnology for diabetes therapy, and a pro...ces to food, supplement, biotechnology and pharmac...Tuesday,s Annual Meeting of Shareholders. ,, At... (Nasdaq: SPEX ) elected the following members to...
(Date:11/19/2009)..., MUNSTER, Germany, November 19 /PRNewswire/... filed on November 16th, 2009 Flibanserin in the,i...inesia in Parkinson,s,disease for orphan drug stat...(EMEA). Levodopa-induced dyskinesia is a complicat...n,s and a serious burden to some of those,affected...
(Date:11/19/2009)... Top LASIK surgeons and recognized ...Trusted LASIK Surgeons made brought their fellow s...ision correction surgery, cataract surgery, and op...merican Academy of Ophthalmology (AAO) annual meet...strom, Dr. Shachar Tauber, Dr. David Huang, MD, Dr...
(Date:11/19/2009)... InSound is honored by latest award... Newark, CA (PRWEB) November 16, 2009 -- P...;invisible” hearing aid from InSound Medical...Award in the Health category.   &nb...igned to reside deep in the hearing canal for up t...
Breaking Biology Technology:Spherix Announces Results of Annual Shareholders Meeting 2Spherix Announces Results of Annual Shareholders Meeting 3Spherix Announces Results of Annual Shareholders Meeting 4MONITORING FORCE GmbH Requests Acknowledgment of Flibanserin as "Orphan Drug" 2Experts Listed at Trusted LASIK Surgeons Bring Peers Up to Date on Vision Correction and Cataract Surgery at Annual AAO Meeting 2Experts Listed at Trusted LASIK Surgeons Bring Peers Up to Date on Vision Correction and Cataract Surgery at Annual AAO Meeting 3Experts Listed at Trusted LASIK Surgeons Bring Peers Up to Date on Vision Correction and Cataract Surgery at Annual AAO Meeting 4Experts Listed at Trusted LASIK Surgeons Bring Peers Up to Date on Vision Correction and Cataract Surgery at Annual AAO Meeting 5Experts Listed at Trusted LASIK Surgeons Bring Peers Up to Date on Vision Correction and Cataract Surgery at Annual AAO Meeting 6Experts Listed at Trusted LASIK Surgeons Bring Peers Up to Date on Vision Correction and Cataract Surgery at Annual AAO Meeting 7Experts Listed at Trusted LASIK Surgeons Bring Peers Up to Date on Vision Correction and Cataract Surgery at Annual AAO Meeting 8Experts Listed at Trusted LASIK Surgeons Bring Peers Up to Date on Vision Correction and Cataract Surgery at Annual AAO Meeting 9Popular Science Awards Lyric “Invisible” Hearing Aid 2009 “Best of What's New” Award 2Popular Science Awards Lyric “Invisible” Hearing Aid 2009 “Best of What's New” Award 3
Other News:
...- Looking to find the beef on Mad Cow Disease? The..., the latest scientific news, and.the most recent ...found.on the World Wide Web (http://w3.aces.uiuc.e... Web site was developed at the University.of Illin...
...s: A photo of Steve Nowicki is available on the Un....19/pub/ .... The filename is Nowicki.... He...ma............... DURHAM, N.C. -- When male song...r song might directly portray their fitness, say D...
COLUMBUS, Ohio -- People who lower their fat or carbohydrate intake.in an attempt to lose weight might be in for a bigger battle than they expected.. A new study found that people who ate low-fat or l
...the American Pediatric Society, the Society for......sociation will present their...findings at the joi...May 1-5. ... Vaccines and vaccination, gen...its effects on children, developmental biology, an...
Web Site Offers Comprehensive Information On Mad Cow Disease 2Birds Sing The 'Story Of Their Lives,' Theorize Duke Biologists 2Birds Sing The 'Story Of Their Lives,' Theorize Duke Biologists 3Birds Sing The 'Story Of Their Lives,' Theorize Duke Biologists 4Eating Less Fat At One Meal May Lead To Higher Fat Intake Later 2Eating Less Fat At One Meal May Lead To Higher Fat Intake Later 3Pediatric Academic Societies Report Progress In Children's Health 2Pediatric Academic Societies Report Progress In Children's Health 3
Scientists from the new interdisciplinary LIMES (Life & Medical Sciences) Centre at the University of Bonn have identified a new gene which could play an important role in the development of diabetes.
... the storerooms of a Venice, Italy, museum, a Univ...n experts are at work on a rare collection, but th...of ancient glassblowers. Instead, the team is coll... collection of fungi in Italy to create an unprece...
...- Expression of two different proteins taken from ...pread of breast cancer to nearby lymph nodes, acco...could help identify at an early stage those patien...he December 15 issue of Cancer Research, the resea...
...I. Brown and Harvard scientists have learned that...s the wings of birds during flight. In an advanced...is tough bit of tissue evolved to become a linchpi...round how limbs became wings is a fascinating qu...
Linchpin discovered in insulin metabolism 2Linchpin discovered in insulin metabolism 3Researchers barcode DNA of Venice museum's vast fungi collection 2Researchers barcode DNA of Venice museum's vast fungi collection 3Researchers barcode DNA of Venice museum's vast fungi collection 4Researchers find two biomarkers with potential to predict breast cancer spread 2Researchers find two biomarkers with potential to predict breast cancer spread 3Shoulder ligament a linchpin in the evolution of flight 2