About Careers Internship MedBlog Contact us
Medindia LOGIN REGISTER
Advertisement

Delivering Replacement SMN1 Gene Found to Extend Survival in Animal Model of Spinal Muscular Atrophy

by Kathy Jones on May 24, 2014 at 3:19 PM
Font : A-A+

 Delivering Replacement SMN1 Gene Found to Extend Survival in Animal Model of Spinal Muscular Atrophy

Researchers have shown in animal models that delivering replacement SMN1 gene directly to the spinal cords can help make up for the insufficient amounts of SMN protein, which leads to inherited neuromuscular disease spinal muscular atrophy (SMA), and thereby extend the survival of the treated animals, a new study published in the journal Human Gene Therapy reveals.

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 AAV9-Survival Motor Neuron 1 for Spinal Muscular Atrophy."

Advertisement

"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.



Source: Eurekalert
Advertisement

Advertisement
News A-Z
A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
What's New on Medindia
World Heart Day in 2022- Use Heart for Every Heart
Anemia among Indian Women and Children Remains a Cause of Concern- National Family Health Survey-5
H1N1 Influenza Prevention in Children: What Parents Need to Know
View all
Recommended Reading
News Archive
Date
Category
Advertisement
News Category

Medindia Newsletters Subscribe to our Free Newsletters!
Terms & Conditions and Privacy Policy.

More News on:
DNA Finger Printing Weaver Syndrome 

Most Popular on Medindia

Sinopril (2mg) (Lacidipine) Blood Donation - Recipients Diaphragmatic Hernia Find a Hospital Hearing Loss Calculator Post-Nasal Drip How to Reduce School Bag Weight - Simple Tips Pregnancy Confirmation Calculator Indian Medical Journals Iron Intake Calculator
This site uses cookies to deliver our services. By using our site, you acknowledge that you have read and understand our Cookie Policy, Privacy Policy, and our Terms of Use
×

Delivering Replacement SMN1 Gene Found to Extend Survival in Animal Model of Spinal Muscular Atrophy Personalised Printable Document (PDF)

Please complete this form and we'll send you a personalised information that is requested

You may use this for your own reference or forward it to your friends.

Please use the information prudently. If you are not a medical doctor please remember to consult your healthcare provider as this information is not a substitute for professional advice.

Name *

Email Address *

Country *

Areas of Interests