Navigation Links
Gene therapy extends survival in large animal models 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 medicine news :

1. The interruption of biological rhythms during chemotherapy worsen its side effects
2. First test of pluripotent stem cell therapy in monkeys is a success
3. Low-dose anticoagulation therapy can be used safely with new design mechanical heart valve
4. New agent may enhance effectiveness of radiotherapy
5. Novel target found for chemotherapy-resistant leukemia cells
6. A form of immune therapy might be effective for multiple myeloma
7. Therapy sought to reduce major risk from minor bleeding that can follow stroke
8. Drug therapy for allergy moves forward
9. Two-lock box delivers cancer therapy
10. WHI reports $37.1B economic return on combined hormone therapy clinical trial
11. Study finds family-based exposure therapy effective treatment for young children with OCD
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Gene therapy extends survival in large animal models of spinal muscular atrophy
(Date:5/6/2016)... Utah (PRWEB) , ... May 06, 2016 , ... ... Step, an online provider of career-focused education and corporate training, and the National ... are pleased to announce Maritza Aquino as the second full-tuition scholarship recipient of ...
(Date:5/5/2016)... ... 2016 , ... TransWipe Volume 3 is a set of 30 professional transition ... preset between two video clips to instantly add a unique transition to any Final ... color and orientation options. TransWipe Volume 3 is a Final Cut Pro X exclusive ...
(Date:5/5/2016)... ... May 05, 2016 , ... The ... electronic cigarettes, requiring e-cigarette manufacturers to submit their products through an arduous federal ... all vaping products that entered the market since February 15, 2007. That would ...
(Date:5/5/2016)... SAN FRANCISCO (PRWEB) , ... May 05, 2016 ... ... in the Unites States to promise successful results with IVF and with egg ... Designed to deliver the most patient-centric and highest quality fertility care, Spring Fertility ...
(Date:5/5/2016)... ... ... Derrin Doty Group has unveiled the latest charity campaign in their ongoing ... Seattle. The insurance provider’s caring team has been so moved by the sudden death ... from the flu, that they have decided to extend their original campaign. Compassionate community ...
Breaking Medicine News(10 mins):
(Date:5/4/2016)... May 4, 2016 According to market ... and Demand Forecast to 2022 - Industry Insights by Slice ... Center and Others)" by P&S Market Research, the global ... 2015, and it is expected to grow at a CAGR ... slice type segment is expected to witness the faster growth, ...
(Date:5/4/2016)... D.C. , May 4, 2016 ... member companies concluded a series of free workshops ... in global requirements for Good Distribution ... of quality assurance which ensures that products are consistently stored, ... the marketing authorization (MA) or product specification. Only a ...
(Date:5/4/2016)... May 4, 2016 ... the  "Global Multiple Myeloma Market and Competitive ... offering.       (Logo: http://photos.prnewswire.com/prnh/20160330/349511LOGO ... and Competitive Landscape Highlights 2016, provides comprehensive ... Myeloma epidemiology, Multiple Myeloma market valuations and ...
Breaking Medicine Technology: