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:11/11/2014)...  Forensicon, Inc., a Chicago -based ... the promotion of Yaniv Schiff from Senior ... Schiff,s new role as Director, he will lead company ... examiners and provide leadership within the company,s digital forensics ... Schiff joined Forensicon in 2006 ...
(Date:11/6/2014)... team of Florida State University biologists could lead to ... and survive environmental swings such as droughts or floods. ... the journal The Plant Cell , sheds light ... is organized in a cell and how plants regulate ... and others are turned off. , "If you understand ...
(Date:11/5/2014)... career to understanding the Earth,s climate and communicating ... deputy director and regional climatologist for the Western ... Redmond has more than three decades of experience ... data to the general public. , At its ... December 15-19, 2014 the American Geophysical Union (AGU) ...
Breaking Biology News(10 mins):Yaniv Schiff Promoted to Director of Digital Forensics at Forensicon, Inc. 2Maize analysis yields whole new world of genetic science 2Nevada climatologist to be honored at AGU Fall Meeting for lifetime of public outreach 2
... New Haven, Conn.Newly created neurons in adults rely on ... and survival before they can communicate with existing neighboring ... of adult neural stem cells to replace brain cells ... researchers report in The Journal of Neuroscience. In ...
... parents helps them better understand the diseases their children ... offering practical advice for preventing infections in their kids ... Complete Guide to Protecting (without Overprotecting) Your Family from ... inside source on germ defense from antibiotics and ...
... of proteins have finally explained why computer models of ... observations. This work could have vast implications in bioengineering ... relationship between structure and function in these basic building ... published as the cover article of the Oct. 16 ...
Cached Biology News:Handbook helps parents deal with childhood infections 2Speed plays crucial role in breaking protein's H-bonds 2Speed plays crucial role in breaking protein's H-bonds 3
(Date:11/22/2014)... 2014 The “Chiral Chromatography Columns ... Material (Metal, Glass, Plastic), by Application [GC, LC ... Forecast to 2018” analyses and studies the major ... America, Europe, Asia, and Rest of the World. ... figures spread through 135 pages and in-depth TOC ...
(Date:11/21/2014)... November 21, 2014 , ...   Mariano Rodríguez es elegid vicepresidente senior ... KLOX está en marcha para comenzar de forma rápida ... heridas de reciente aprobación en Europa   , ... complace al anunciar los siguientes nombramientos: Todd ...
(Date:11/21/2014)... (PRWEB) November 20, 2014 Author Matthew ... Walk to the Other Side” published as a ... Finalist in the “Fiction: Short Story” category of the ... Keen, President and CEO of USA Book News, said ... and independent publishers, including Simon & Schuster, Penguin, John ...
(Date:11/21/2014)... Fayetteville, Arkansas (PRWEB) November 20, 2014 ... partners, Pinnacle Ozone Solutions, LLC (Pinnacle), and PCI, ... equipment, announced that they have been selected to ... City of Fayetteville, Arkansas Paul R. Noland Wastewater ... feature BlueInGreen’s patented HyDOZ® gas dissolution ...
Breaking Biology Technology:Chiral Chromatography Columns Market worth $87.8 Million by 2018 - New Research Report by MarketsandMarkets 2Chiral Chromatography Columns Market worth $87.8 Million by 2018 - New Research Report by MarketsandMarkets 3Chiral Chromatography Columns Market worth $87.8 Million by 2018 - New Research Report by MarketsandMarkets 4Chiral Chromatography Columns Market worth $87.8 Million by 2018 - New Research Report by MarketsandMarkets 5KLOX Technologies anuncia sus nombramientos ejecutivos 2KLOX Technologies anuncia sus nombramientos ejecutivos 3KLOX Technologies anuncia sus nombramientos ejecutivos 4KLOX Technologies anuncia sus nombramientos ejecutivos 5Mystic Ink 's Short Story Collection “A Short Walk to the Other Side” Honoring Ray Bradbury Praised As Award-Winning Finalist In USA Best Book Awards 2Mystic Ink 's Short Story Collection “A Short Walk to the Other Side” Honoring Ray Bradbury Praised As Award-Winning Finalist In USA Best Book Awards 3Mystic Ink 's Short Story Collection “A Short Walk to the Other Side” Honoring Ray Bradbury Praised As Award-Winning Finalist In USA Best Book Awards 4City of Fayetteville Leads Nation with Innovative Ozone Technology 2City of Fayetteville Leads Nation with Innovative Ozone Technology 3City of Fayetteville Leads Nation with Innovative Ozone Technology 4
... Mass., June 2 Cynosure, Inc. (Nasdaq: ... of light-based aesthetic treatment systems, today announced that President and Chief Executive ... present at the following investor conferences in New York City this month: ... Gabelli & Company Healthcare Symposium , Tuesday, ...
... ... ClearTrial will lead panel of adaptive trial experts offering practical guidelines ... become more prevalent, their operational planning challenges often remain overlooked in ... on the initial trial design, ranging from understanding the potential for ...
... Micromet, Inc. (Nasdaq: MITI ), a biopharmaceutical company developing ... diseases, today announced that it will host a webcast on June ... and 6th at the 14th Congress of the European Hematology Association. ... www.micromet-inc.com . , , Forum: ...
Cached Biology Technology:Cynosure Announces Presentations at Upcoming Investor Conferences 2ClearTrial to Chair Session at DIA 45th Annual Meeting on "Effective Operational Planning for Adaptive Clinical Trials" 2ClearTrial to Chair Session at DIA 45th Annual Meeting on "Effective Operational Planning for Adaptive Clinical Trials" 3Micromet to Host Conference Call and Webcast to Discuss Blinatumomab Data Presented at EHA on June 8, 2009 2Micromet to Host Conference Call and Webcast to Discuss Blinatumomab Data Presented at EHA on June 8, 2009 3Micromet to Host Conference Call and Webcast to Discuss Blinatumomab Data Presented at EHA on June 8, 2009 4
Mouse polyclonal antibody to TNPO2 - transportin 2 (importin 3, karyopherin beta 2b)...
... HDAC4 - ChIP Grade HDAC4 is ... histone deacetylases, which consists of 1084 amino ... similar to the deacetylase domain of yeast ... the nucleus and cytoplasm in a process ...
...
Mouse monoclonal antibody to PDK2 - pyruvate dehydrogenase kinase, isoenzyme 2...
Biology Products: