Navigation Links
Treatment approach to human Usher syndrome: Small molecules ignore stop signals
Date:7/1/2011

signal in the mutated USH1C gene to be ignored, thus resulting in continuing protein synthesis and the formation of the functional genetic product in cell and organ cultures. In addition to its ability to cause readthrough of stop signals, the active agent PTC124 has also been demonstrated to be highly compatible with murine and human retina cultures. Moreover, the team managed for the first time to demonstrate readthrough of an eye mutation codon in vivo.

"PTC124 is already being tested in clinical trials for its efficacy in treating other diseases involving nonsense mutations, such as cystic fibrosis and Duchenne muscular dystrophy. We therefore hope that this treatment approach will soon be ready for use in Usher syndrome patients," explains Dr Kerstin Nagel-Wolfrum.

Currently putting the finishing touches on his doctoral thesis, Tobias Goldmann is comparing the efficiency of the readthrough rate and the biocompatibility of other molecules that induce the readthrough of nonsense mutations. The focus is particularly on modified aminoglycosides, i.e. derivatives of commercially available and clinically tested antibiotics. These are being designed and synthesized by an Israeli cooperation partner, Professor Timor Bassov of the Haifa Technicon, and have already been successfully used by researchers in Mainz for readthrough of nonsense mutations in Usher genes. In addition to conducting further preclinical studies of the ocular applications of these new substances, the Usher laboratory in Mainz is planning to use this new method of treating this specific form of Usher syndrome in hospital patients as soon as possible.

The translational biomedical research on readthrough of nonsense mutations aimed at developing a treatment for Usher syndrome is being funded by the FAUN foundation and the "Syscilia" project of the Seventh Framework Program of the European Union. It is part of the Research Training Group 1044 "Developmental and Disease-Ind
'/>"/>

Contact: Dr. Kerstin Nagel-Wolfrum
nagelwol@uni-mainz.de
49-613-139-20131
Johannes Gutenberg Universitaet Mainz
Source:Eurekalert

Page: 1 2 3

Related biology news :

1. IOF calls for improved strategies to close the treatment gap and reduce future burden of fractures
2. Scripps Research scientists awarded $2.2 million to develop treatment for multi-drug addiction
3. Potential treatment for deadly E. coli disease
4. Research reveals effectiveness of seizure treatments for children with autism
5. Therapies to improve biochemical functions hold promise as treatments for autism
6. Cancer drug holds promise as first treatment for common, inherited dementia
7. Arthritis patients taking newer treatments do not have an overall increased cancer risk
8. Undertreatment of cardiovascular disease in rheumatoid arthritis patients following a heart attack
9. Several abstracts on gout reveal true burden of disease and highlight promising new treatments
10. Targeted adalimumab treatment can optimize long-term outcomes for patients with early RA
11. Recycling of Alzheimers proteins could be key to new treatments
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/9/2015)... 9, 2015  Lintec of America recently announced an ... nanotube (CNT) macrostructures, including sheets, yarns and ribbons, developed ... Leveraging the vast industrial resources of the global ... , Lintec of America is forming the Nano-Science and ... , focusing on scaling up the manufacturing and commercialization ...
(Date:2/5/2015)... Conn. , Jan. 26, 2015 NXT-ID, Inc. ... the "Company"), a biometric authentication company focused on the ... new corporate website ( http://www.nxt-id.com/ ) as part of ... smart wallet. The Company launched its new consumer website ... Gino Pereira , CEO of NXT-ID said, "Our new ...
(Date:2/5/2015)... Research and Markets ( http://www.researchandmarkets.com/research/6lngtl/global_biometrics ... "Global Biometrics Market (2014-2020): Market Forecast By Technologies, ... to their offering. , In ... is anticipated to overtake North America ... spending towards IT security, government biometric based projects ...
Breaking Biology News(10 mins):Lintec announces exclusive license to commercialize innovative carbon nanotube technology from the University of Texas at Dallas 2Lintec announces exclusive license to commercialize innovative carbon nanotube technology from the University of Texas at Dallas 3Lintec announces exclusive license to commercialize innovative carbon nanotube technology from the University of Texas at Dallas 4NXT-ID, Inc. Launches New Corporate Website As Part Of Ongoing 2015 Branding Initiatives For Wocket Smart Wallet 2NXT-ID, Inc. Launches New Corporate Website As Part Of Ongoing 2015 Branding Initiatives For Wocket Smart Wallet 3Global Biometrics Market (2014-2020): Market Forecast By Technologies, Applications, End Use, Regions and Countries 2Global Biometrics Market (2014-2020): Market Forecast By Technologies, Applications, End Use, Regions and Countries 3
... may not be the only organisms endangered by some food ... health risks to humans who eat meat from chickens that ... the April 9 issue of Chemical & Engineering News, the ... Roxarsone, the most common arsenic-based additive used in chicken feed, ...
... As people age, they may have to exercise even harder ... suggestion of a report in the February issue of the ... signal that gives muscles a kind of metabolic boost in ... researchers found that so-called AMP-activated protein kinase (AMPK) slows down ...
... to repair the human body, replacing defective and worn ... the body does not always accept such substitutes seamlessly. ... simply changing the surface texture of implants can dramatically ... materials. , Two recent experiments have focused on ...
Cached Biology News:Power-boosting signal in muscle declines with age 2Nanotextured implant materials: blending in, not fighting back 2Nanotextured implant materials: blending in, not fighting back 3
(Date:3/4/2015)... 2015 AcelRx Pharmaceuticals, Inc. (Nasdaq: ... on the development and commercialization of innovative therapies ... today announced that it will release fourth quarter ... on Monday, March 9th, 2015. AcelRx management will ... Eastern Time (1:30 p.m. Pacific Time) on March ...
(Date:3/4/2015)... PARIS , March 4, 2015 ... de Paris in December 2011, ... study of yttrium-90 resin microspheres versus sorafenib in advanced ... results expected late 2016.   SARAH, a ... liver cancer (hepatocellular carcinoma, or HCC) has completed patient ...
(Date:3/3/2015)... Therapeutics, Inc. (NASDAQ: HALO ) will be presenting ... Miami, FL on March 10, 2015 at 1:05 ... Financial Officer, will provide a corporate overview. The ... corporate website at www.halozyme.com , and a recording will ... access the live webcast, please log on to Halozyme,s website ...
(Date:3/3/2015)... , Mar. 03, 2015 Research and Markets ... the "Gene Therapy Market, 2015 - 2025" ... Therapy Market, 2015-2025" report provides an extensive study on ... research has been carried out in this field for ... therapies (four available in Asian markets; one approved in ...
Breaking Biology Technology:AcelRx Pharmaceuticals to Hold Annual 2014 Financial Results Conference Call and Webcast on Monday, March 9th, 2015 2AcelRx Pharmaceuticals to Hold Annual 2014 Financial Results Conference Call and Webcast on Monday, March 9th, 2015 3Treatment of Primary Liver Cancer: SARAH Study Completes Enrolment, Results Expected Late 2016 2Treatment of Primary Liver Cancer: SARAH Study Completes Enrolment, Results Expected Late 2016 3Treatment of Primary Liver Cancer: SARAH Study Completes Enrolment, Results Expected Late 2016 4Treatment of Primary Liver Cancer: SARAH Study Completes Enrolment, Results Expected Late 2016 5Halozyme Therapeutics To Present At The Barclays 2015 Global Healthcare Conference 2Global Gene Therapy Market Report 2015-2025 - Extensive Study on the Marketed and Pipeline Gene Therapies 2Global Gene Therapy Market Report 2015-2025 - Extensive Study on the Marketed and Pipeline Gene Therapies 3Global Gene Therapy Market Report 2015-2025 - Extensive Study on the Marketed and Pipeline Gene Therapies 4Global Gene Therapy Market Report 2015-2025 - Extensive Study on the Marketed and Pipeline Gene Therapies 5Global Gene Therapy Market Report 2015-2025 - Extensive Study on the Marketed and Pipeline Gene Therapies 6Global Gene Therapy Market Report 2015-2025 - Extensive Study on the Marketed and Pipeline Gene Therapies 7
... GERMANTOWN, Maryland and HILDEN, Germany ... QGEN ; Frankfurt , Prime Standard:,QIA) today announced ... Institute,for Product Quality (ifp), pursuant to which QIAGEN has ... by the Berlin -based specialized,laboratory center for food ...
... ... and industry leaders from around the globe to discuss pain management, preclinical and clinical development ... ... Pain Therapeutics Summit , set to take place on October 4th-5th, 2010 in Philadelphia, has ...
... , May 25 Regado Biosciences, Inc., ... aptamers with active control agents, today announced issuance of ... new patent includes coverage for RB007, the modulator or ... REG1 and REG2 anticoagulation systems.  The allowed claims in ...
Cached Biology Technology:QIAGEN Acquires Comprehensive Portfolio of Molecular Food Safety Tests 2QIAGEN Acquires Comprehensive Portfolio of Molecular Food Safety Tests 3QIAGEN Acquires Comprehensive Portfolio of Molecular Food Safety Tests 4QIAGEN Acquires Comprehensive Portfolio of Molecular Food Safety Tests 5QIAGEN Acquires Comprehensive Portfolio of Molecular Food Safety Tests 6QIAGEN Acquires Comprehensive Portfolio of Molecular Food Safety Tests 7Scientific and Business Leaders to Discuss Challenges Facing Developers of Pain Therapeutics at Arrowhead's 4th Annual Pain Therapeutics Summit 2Regado Biosciences, Inc. Continues to Expand Its Patent Portfolio By Announcing Issuance of a New Patent for Its Product Candidates, the REG1 and REG2 Anticoagulation Systems 2Regado Biosciences, Inc. Continues to Expand Its Patent Portfolio By Announcing Issuance of a New Patent for Its Product Candidates, the REG1 and REG2 Anticoagulation Systems 3
Collected from animals that are 16 to 18 months old. Sterile, Cell culture tested, ID clarifier: USA Origin...
... 20-HETE is a cytochrome P450 (CYP450) metabolite ... the renal and cerebral vasculature. In rat ... mediates pressure-induced autoregulatory vasoconstriction. 20-HETE is excreted ... of free 20-HETE (20-40 pg/ml in human ...
... GPS-M Mutagenesis System enables you to insert ... into,your target DNA by in vitro transposition. ... Tn7-based,transposon randomly into the DNA target. Target ... or purified,chromosomal DNA, depending on your interests ...
A set of five peptides which can be used to calibrate a Matrix-assisted laser desorption/ionization time-of-flight (MALDI-TOF) or Electrospray Ionization (ESI) mass spectrometer....
Biology Products: