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:5/6/2017)... -- RAM Group , Singaporean based technology ... biometric authentication based on a novel  quantum-state ... perform biometric authentication. These new sensors are based on a ... Group and its partners. This sensor will have widespread ... security. Ram Group is a next generation sensor ...
(Date:4/17/2017)... , April 17, 2017 NXT-ID, Inc. (NASDAQ: ... announces the filing of its 2016 Annual Report on Form 10-K ... Commission. ... 10-K is available in the Investor Relations section of the Company,s ... the SEC,s website at http://www.sec.gov . 2016 Year ...
(Date:4/11/2017)... 2017 Research and Markets has announced the ... to their offering. ... eye tracking market to grow at a CAGR of 30.37% during ... Market 2017-2021, has been prepared based on an in-depth market analysis ... and its growth prospects over the coming years. The report also ...
Breaking Biology News(10 mins):
(Date:10/11/2017)... ... October 11, 2017 , ... At ... Purple announced Dr. Suneel I. Sheikh, the co-founder, CEO and chief research scientist ... has been selected for membership in ARCS Alumni Hall of Fame . ...
(Date:10/11/2017)... BioMarketing, a leading provider of patient support solutions, has announced ... network, which will launch this week. The VMS CNEs will ... to enhance the patient care experience by delivering peer-to-peer education ... professionals to help women who have been diagnosed and are ... ...
(Date:10/10/2017)... ... 2017 , ... San Diego-based team building and cooking events company, Lajollacooks4u, has ... The bold new look is part of a transformation to increase awareness, appeal to ... period. , It will also expand its service offering from its signature gourmet cooking ...
(Date:10/10/2017)... Oct. 10, 2017 SomaGenics announced the receipt ... to develop RealSeq®-SC (Single Cell), expected to be the ... RNAs (including microRNAs) from single cells using NGS methods. ... need to accelerate development of approaches to analyze the ... "New techniques for measuring levels of mRNAs in ...
Breaking Biology Technology: