Navigation Links
Caltech researchers show efficacy of gene therapy in mouse models of Huntington's disease
Date:10/30/2009

bodycalled Happ1was an unqualified success, restoring motor and cognitive function to the mice, and reducing neuron loss as well as toxic protein accumulation. And in one model, it increased both body weight and life span.

Happ1 targets an amino-acid sequence unique to the Htt protein that is rich in the amino acid proline. Because of this, the action of Happ1 is expected to be extremely specific. "Our studies show that the use of intrabodies can block the parts of mutant huntingtin that cause its toxicity without affecting the wildtype, or normal, huntingtinor any other proteins," says Patterson. In other words, he says, this has the potential to be the kind of "silver-bullet therapy" that many medical researchers look for.

This sort of research is of particular importance in the treatment of Huntington's disease, says Patterson. Despite the fact that this disorder has a single-gene origin, current treatments tend to address the symptoms of the disease, not its cause. That means it is currently impossible to prevent the disease from doing significant damage in the first place.

What's the next step in pursuit of this goal? "We need to improve the efficacy of the intrabody," Patterson says, "and we need to build a viral vector that can be controlledinduced and turned offin case of unexpected side effects. This is a general goal shared by all types of experimental gene therapies."


'/>"/>

Contact: Lori Oliwenstein
lorio@caltech.edu
626-395-3631
California Institute of Technology
Source:Eurekalert

Page: 1 2

Related biology news :

1. Caltech scientists create robot surrogate for blind persons in testing visual prostheses
2. Caltech scientists get detailed glimpse of chemoreceptor architecture in bacterial cells
3. Caltech neuroscientists find brain region responsible for our sense of personal space
4. Caltech researchers explore how cells reconcile mixed messages in decisions about growth
5. Caltech scientists reveal how neuronal activity is timed in brains memory-making circuits
6. Caltech scientists show why anti-HIV antibodies are ineffective at blocking infection
7. Caltech scientists control complex nucleation processes using DNA origami seeds
8. Caltech researchers train computers to analyze fruit-fly behavior
9. Caltech and UCSD researchers shed light on how proteins find their shapes
10. Caltech researchers help unlock the secrets of gene regulatory networks
11. Caltech researchers get first look at how groups of cells coordinate their movements
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/18/2015)... , June 18, 2015 This report ... Million by the following Product Segments, which includes Immunoassay ... Chemiluminescence Immunoassay Systems, Radio Immunoassay Systems, and Nephelometric Immunoassay ... US, Canada , Japan ... Asia-Pacific , Latin America , ...
(Date:6/17/2015)... Deutschland und GERMANTOWN, Maryland , June ... ®   Produktlinie mit DNA-Tests verbessert den ... (NASDAQ: QGEN ; Frankfurt Prime Standard: QIA) hat ... STR-Testkits der Produktlinie Investigator ® ... Markt eingeführt. Die neuen Kits zur Erstellung genetischer Fingerabdrücke ...
(Date:6/17/2015)... , June 17, 2015  Synaptics Inc. (NASDAQ: ... solutions, today announced that Xiaomi, one of the ... ® ClearPad ® family of capacitive ... integrated circuits (DDICs) for its latest smartphones, the ... ClearPad for full in-cell display solutions and DDICs ...
Breaking Biology News(10 mins):Global Immunoassay Systems Market 2Global Immunoassay Systems Market 3Global Immunoassay Systems Market 4Global Immunoassay Systems Market 5Global Immunoassay Systems Market 6Global Immunoassay Systems Market 7Global Immunoassay Systems Market 8Global Immunoassay Systems Market 9Global Immunoassay Systems Market 10Global Immunoassay Systems Market 11Global Immunoassay Systems Market 12Global Immunoassay Systems Market 13Global Immunoassay Systems Market 14Global Immunoassay Systems Market 15Global Immunoassay Systems Market 16Global Immunoassay Systems Market 17Global Immunoassay Systems Market 18Global Immunoassay Systems Market 19Global Immunoassay Systems Market 20Global Immunoassay Systems Market 21Global Immunoassay Systems Market 22Global Immunoassay Systems Market 23Global Immunoassay Systems Market 24Global Immunoassay Systems Market 25Global Immunoassay Systems Market 26Global Immunoassay Systems Market 27Global Immunoassay Systems Market 28Global Immunoassay Systems Market 29QIAGEN bringt in den USA neue Kits für die forensische DNA-Analyse auf den Markt 2QIAGEN bringt in den USA neue Kits für die forensische DNA-Analyse auf den Markt 3QIAGEN bringt in den USA neue Kits für die forensische DNA-Analyse auf den Markt 4QIAGEN bringt in den USA neue Kits für die forensische DNA-Analyse auf den Markt 5QIAGEN bringt in den USA neue Kits für die forensische DNA-Analyse auf den Markt 6Synaptics Touchscreen and Display Driver IC Solutions Power Xiaomi's Latest Flagship Smartphones 2Synaptics Touchscreen and Display Driver IC Solutions Power Xiaomi's Latest Flagship Smartphones 3
... clear skies and smooth sailing for oceanic vessels has ... to air pollution and greenhouse gas emissions that impact ... James Winebrake and James Corbett examines "Emission Tradeoffs among ... Oil, Marine Gas Oil, and Marine Diesel Oil," in ...
... The 2009 Force Protection Equipment,Demonstration (FPED VII) will ... and Marine Corps Base, Quantico, VA, next May ... War on Terror., The demonstration is the ... commercial off-the-shelf equipment solutions to current,and evolving force ...
... new paper by a team of researchers led by ... the University of Notre Dame, provides important new insights ... wall. The cell wall is a critically important ... is a rigid entity encasing the bacterium, and antibiotics ...
Cached Biology News:Fuel emissions from marine vessels remain a global concern 22009 Force Protection Equipment Demonstration Set for Next May 19-21 2
(Date:7/3/2015)... ... July 03, 2015 , ... The global failure analysis market is ... to 2020. The bioscience market is expected to grow at the highest CAGR of ... analysis equipment in the research labs and academic institutions for root cause analysis of ...
(Date:7/3/2015)... Forskere kan ansøge ... http://www.openinnovationinscience.at om efteruddannelsesprogrammet "Lab for Open ... Wien .  ... og videnskabsmænd foretaget af Ludwig Boltzmann Gesellschaft ... sundhedsvidenskaben manglen på incitamenter til at undersøge ...
(Date:7/2/2015)... July 2, 2015 Amici Procurement ... procurement company Amici Procurement has chosen Microsoft Cloud Solution ... move to Office 365.      (Logo: http://photos.prnewswire.com/prnh/20121126/579634) ... to medium-sized companies in the bio-tech sector with a ... the purchasing cycle to help them obtain best value. ...
(Date:7/1/2015)... PARIS , July 1, 2015 ... key developer of biotech crops, today announce a ... generations of insect control traits. "Growers ... the realities of advancing insect resistance. We are ... insect control technology with Genective,s capability in developing ...
Breaking Biology Technology:Failure Analysis Market to Grow at 7.52% CAGR to 2020 2Failure Analysis Market to Grow at 7.52% CAGR to 2020 3Failure Analysis Market to Grow at 7.52% CAGR to 2020 4Failure Analysis Market to Grow at 7.52% CAGR to 2020 5Lancering af verdens første åbne innovationsprogram til uddannelse af forskere 2Lancering af verdens første åbne innovationsprogram til uddannelse af forskere 3Biotech Procurement Company Moves to Office 365 with iomart 2AgBiome and Genective collaborate to create novel insect-resistant crops 2
... Laurie Benson may be as different as, well, New York ... women entrepreneurs, including those operating in the technology space. , ... can see, but the result may be a largely untapped ... their business to the next level. Specifically, the million-dollar level. ...
... pace of change sparked by the information technology industry ... every business challenge, but has IT really come through ... a recent Wisconsin technology conference, the results are mixed ... Development Systems , brought together more than 300 corporate ...
... - Brady Corp. , a manufacturer of ... appointment of Robert Tatterson to the position of chief ... and product innovation, materials development, and chemical engineering, will ... and Asia. He will report to company President and ...
Cached Biology Technology:Women entrepreneurs reach for $1M level 2Women entrepreneurs reach for $1M level 3Are businesses getting what they need from IT? 2Are businesses getting what they need from IT? 3Brady appoints GE efficiency expert as CTO 2
...
...
...
... volume pipetting is increasingly essential for ... DNA sequencing and other biochemical applications. ... and precision unparalleled in the industry. ... of all aspirated,and dispensed volumes , ...
Biology Products: