Navigation Links
Gene therapy extends survival in an animal model of spinal muscular atrophy
Date:5/22/2014

New Rochelle, NY, May 22, 2014To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords of animal models of SMA. A new study demonstrating that enough copies of the SMN1 gene can be delivered to the spinal cord motor neurons to extend the survival of the treated animals is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website.

Marco Passini and coauthors from Genzyme (Framingham, MA), University of California San Francisco, Emory University School of Medicine (Atlanta, GA), and Georgetown University Medical Center (Washington, DC) used an adeno-associated viral vector as the delivery vehicle to transport copies of the SMN1 gene into motor neurons in the spinal cord via intrathecal delivery. They report on the effectiveness of restoring the levels of functional SMN protein in normal pig and non-human primate SMA models that would predict efficacy based on gene transfer with the same vector in an authentic mouse model of SMA in the article "Translational Fidelity of Intrathecal Delivery of Self-Complementary AAV9Survival Motor Neuron 1 for Spinal Muscular Atrophy."

"This is a very promising and thorough set of preclinical studies that supports rapid translation to the clinic," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.


'/>"/>

Contact: Kathryn Ruehle
kruehle@liebertpub.com
914-740-2100
Mary Ann Liebert, Inc./Genetic Engineering News
Source:Eurekalert  

Page: 1

Related biology news :

1. Novel RNAi therapy silences mutated Huntingtons disease gene and reduces symptoms
2. Phase I data suggest PLX3397 is a potential therapy for patients with advanced PVNS
3. Chemotherapy timing is key to success
4. Soy sauce molecule may unlock drug therapy for HIV patients
5. Autologous stem cell therapy improves motor function in chronic stroke victims
6. Loss of memory in Alzheimers mice models reversed through gene therapy
7. Radiation therapy for cervical cancer increases risk for colorectal cancer
8. Friedreichs ataxia -- an effective gene therapy in an animal model
9. New Penn-designed gel allows for targeted therapy after heart attack
10. Natural plant compounds may assist chemotherapy
11. Immunotherapy data heralds new era of lung cancer treatment
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Gene therapy extends survival in an animal model of spinal muscular atrophy
(Date:7/30/2014)... of cells types. Researchers would like to understand how to ... that make up the organs and other structures of living ... of sugars that dangle from proteins on surfaces of cells. ... Diego, has created synthetic molecules that can stand in for ... direct the process, they report in the Journal of ...
(Date:7/29/2014)... New research by academics in the University of Bristol,s ... we take liquid calories into account when planning meals. ... for the Study of Ingestive Behavior Conference (SSIB 2014) ... August], argues that we do. , The team was ... the School of Experimental Psychology. , As part of ...
(Date:7/29/2014)... Research and Markets  has announced the addition of ... report to their offering. ... refers to applications and technologies that are used for ... company,s operations. BI help organizations to acquire comprehensive knowledge ... on sales, production, and internal operations. ...
Breaking Biology News(10 mins):Global SaaS-based Business Intelligence (BI) Market 2014-2018 2
... membranes could help phytoplankton cope with nutrient scarcity in the ... of the National Oceanography Centre has shown that a species ... of its cell membranes in response to changes in nutrient ... for nutrient cycling and the population dynamics of phytoplankton in ...
... JUPITER, FL, December 16, 2010 Scientists from ... shown that prions, bits of infectious protein that can ... (BSE) or "mad cow disease," have the ability to ... In this regard, although they lack DNA and RNA, ...
... Researchers funded by the Biotechnology and Biological Sciences Research ... clue that may help to explain why the earliest evidence ... ago, when atmospheric oxygen levels on the planet rose sharply ... The team, led by Professor Chris Schofield, has found that ...
Cached Biology News:Efficient phosphorus use by phytoplankton 2Scripps Research scientists show prions mutate and adapt to host environment 2Scripps Research scientists show prions mutate and adapt to host environment 3550 million years ago rise in oxygen drove evolution of animal life 2
(Date:7/30/2014)... STONY BROOK, NY (PRWEB) July 30, 2014 ... (OTCQB: APDN), (Twitter: @APDN), a biotechnology firm that provides ... Dr. Michael E. Hogan, Ph.D. Vice President of Life ... in mid-September. , Dr. Hogan will lead ... to form business relationships with biotech and healthcare customers, ...
(Date:7/30/2014)... Scranton, PA (PRWEB) July 30, 2014 ... Flametec® family of products, designed to meet Factory Mutual ... cleanroom equipment materials such as wet benches, process tools, ... to worker safety and damage to semiconductor wafers used ... provides a flame-retardant PVC solution with excellent workability ...
(Date:7/30/2014)... , July 30, 2014  Orexigen Therapeutics, Inc. ... received the Day 180 List of Outstanding Issues (LOI) ... for Human Use (CHMP) for the NB32 Marketing Authorization ... SR) is an investigational drug candidate being evaluated for ... earlier Day 120 time point were adequately addressed by ...
(Date:7/30/2014)... , July 30, 2014 Researchers often ... interrogate biology, but such devices can do much ... As such, researchers have looked to patterned ... vitro metabolic engineering to characterize – and ... New devices are envisioned to recreate animal and ...
Breaking Biology Technology:Dr. Michael E. Hogan, Ph.D., Joins Applied DNA Sciences as Vice President, Life Sciences 2Dr. Michael E. Hogan, Ph.D., Joins Applied DNA Sciences as Vice President, Life Sciences 3Vycom Announces Flametec® Thermax PVC (FM4910 Listed) For a Variety of Clean Room Applications with Excellent Workability and Aesthetics 2Orexigen Receives CHMP Day 180 List of Outstanding Issues 2Orexigen Receives CHMP Day 180 List of Outstanding Issues 3UMD Researchers Bridge Gap between Microelectronics, Biological Systems 2UMD Researchers Bridge Gap between Microelectronics, Biological Systems 3UMD Researchers Bridge Gap between Microelectronics, Biological Systems 4
... Foundation for Angelman Syndrome Therapeutics (FAST) announces it will ... treatment for the rare genetic disorder, Angelman Syndrome (AS).  ... study will examine if the off label administration ... with Angelman Syndrome. Minocycline, an FDA approved antibiotic, is ...
... Feb. 14, 2012  Milo Biotechnology will receive $250,000 ... assistance and some capital into early stage Northeast ... a therapy to increase muscle strength and improve ... Milo is the 61st company in JumpStart,s portfolio. ...
... Gen-Probe Incorporated (NASDAQ: GPRO ) today reported ... in the quarter grew 16%, to a new record of ... to a new high of $0.72. "Gen-Probe finished ... quarter while clinical diagnostics revenues continued their solid growth," said ...
Cached Biology Technology:Foundation for Angelman Syndrome Therapeutics (FAST) Funds Human Clinical Trial 2Foundation for Angelman Syndrome Therapeutics (FAST) Funds Human Clinical Trial 3JumpStart Invests $250,000 in Milo Biotechnology 2Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 2Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 3Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 4Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 5Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 6Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 7Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 8Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 9Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 10Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 11Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 12Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 13Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 14Gen-Probe Reports Financial Results for the Fourth Quarter of 2011 15
Automated Cell Culture Flask...
... system, 100-240 V, is an optional module ... automates delivery of up to 20 L ... reader. The HTF system automatically draws sheath ... to constantly maintain a reservoir of pressurized ...
... every research laboratory, the revolutionary Luminex 100 ... inexpensively and in real time enhancing your ... system includes the Luminex 100, the XY ... technology which enables you to simultaneously assay ...
... routine binary separations. Excellent detection is provided ... variable wavelength detector. Ultra-fine control of the ... highly accurate and precise, high pressure dual piston ... may be plotted on a printer or an ...
Biology Products: