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:8/18/2017)... ... 2017 , ... Mr. Noppadon Pakprot, Deputy Governor for Tourism ... and Wellness Tourism Showcase 2017 yesterday, which unveiled the latest “Functional & Regenerative ... and Business at TAT said, “Thailand has long been recognized as a world ...
(Date:8/18/2017)... Lynchburg, VA (PRWEB) , ... August 18, 2017 , ... ... to the Democratic Republic of Congo (DRC) thanks to an ambitious venture that conjoined ... as well as the generous support of the Liberty community. These shoes will save ...
(Date:8/18/2017)... , ... ... ... For Immediate Release                Contact: ... Shows Young Women Seek Sex and Relationship Advice from their ...
(Date:8/18/2017)... ... August 18, 2017 , ... ... a highly specialized asset-light logistics provider of complex transport solutions for mission-critical ... to purchase Unitrans International Corporation, a division of Roadrunner Transportation Systems, Inc. ...
(Date:8/18/2017)... NY (PRWEB) , ... August 18, 2017 , ... ... of “Vision & Hearing” campaign, advocating for active, healthy lifestyles and highlighting the ... available for individuals with hearing impairments and shares the latest innovations in hearing ...
Breaking Medicine News(10 mins):
(Date:8/17/2017)... , Aug. 17, 2017 Bio-Ess Laboratories, LLC Chief Science Officer, ... titer and present new data on Cell-Ess® Universal Titer Boost ... Boston, MA August 21-25.  The Bioprocessing Summit convenes more ... challenges. ... ...
(Date:8/15/2017)... 2017  AOTI Inc. announced today that its fully owned ... opened a New York City Office in Yonkers, New ... Topical Wound Oxygen (TWO 2 ) homecare therapy. This new East ... Health Care (ACHC) under the company,s DMEPOS accreditation for Home/Durable Medical ... Advanced Oxygen ...
(Date:8/8/2017)...   Second-quarter 2017 revenues of ... share from continuing operations ... to $110 million Second-quarter ... million Second-quarter 2017 adjusted ... 8 percent to $0.93 ...
Breaking Medicine Technology: