Navigation Links
Therapy slows onset and progression of Lou Gehrig's disease, study finds
Date:9/9/2013

at is to deliver the drug directly into the cerebrospinal fluid (CSF), which would reduce the amount of SOD1 suppression in cells outside the brain and reduce immune system exposure to AAV9elements that would add weight to an argument for studying the drug in humans.

Injections directly into CSF cannot be done easily in mice, so the team took the study a crucial step further by injecting AAV9-SOD1-shRNA into the CSF of healthy nonhuman primates. The results were just as the team hopedthe amount of gene expression dropped by as much as 90 percent in motor neurons and nearly 70 percent in astrocytes and no side effects were reported, laying the groundwork towards moving to human clinical trials.

"We have a vast amount of work to do to move this toward a clinical trial, but we're encouraged by the results to date and our team at Nationwide Children's and our outstanding collaborators are fully committed to making a difference in this disease," Dr. Kaspar said.

The findings could impact other studies underway in Dr. Kaspar's lab, including research on Spinal Muscular Atrophy, an often fatal genetic disease in infants and children that can cause profoundly weakened muscles in the arms and legs and respiratory failure.

"This research provides further proof of targeting motor neurons and glial cells throughout the entire spinal cord for treatment of Spinal Muscular Atrophy and other degenerative diseases of the brain and spinal cord, through a less invasive manner than direct injections," said Dr. Kaspar, who also is an associate professor of pediatrics and neurosciences at The Ohio State University College of Medicine.


'/>"/>

Contact: Gina Bericchia
Gina.Bericchia@NationwideChildrens.org
614-355-0495
Nationwide Children's Hospital
Source:Eurekalert

Page: 1 2 3 4

Related biology news :

1. Physiotherapy in Singapore Clinic Urbanrehab Pte Ltd Announces the Opening of an Additional Location
2. Chemotherapy before radiotherapy for testicular cancer could reduce long-term side-effects
3. ITN type 1 diabetes study identifies subset of patients with strong response to therapy
4. Combined therapy could repair and prevent damage in Duchenne muscular dystrophy
5. Study suggests way to fight therapy resistant leukemia by blocking DNA repair
6. An effective initial polytherapy for infantile spasms
7. UCLA stem cell gene therapy for sickle cell disease advances toward clinical trials
8. Neuroblastoma: Autophagy protects from chemotherapy
9. Dendritic cell therapy improves kidney transplant survival, Pitt team says
10. No danger of cancer through gene therapy virus
11. An article in Cell reveals a new resistance mechanism to chemotherapy in breast and ovarian cancer
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/1/2015)... 1, 2015   Medisafe ™, the leading global ... iOS and Android smartphones and ... into the platform to allow patients to track and ... first time, patients will be able to visualize in ... important biometrics, such as glucose levels and blood pressure. ...
(Date:3/26/2015)... , March 26, 2015 The ... social, recreation and athletic club, today announced it has ... allow freedom of movement for members and staff, while ... a comprehensive process, we selected FST,s IMID Access system ... convenience for our members and staff, in addition to ...
(Date:3/24/2015)... , March 24, 2015   Crossmatch ™, ... today announced that its U.are.U ® 4500 ... Goorin Bros . to achieve PCI compliance ... secures access to the POS terminal, protecting the ... of data breaches. With one-third ...
Breaking Biology News(10 mins):Medisafe Announces Launch of Biometric Integration, Enabling Patients to Stay Adherent to Their Meds and See Tangible Health Results 2Granite Club First Private Club in North America to Implement FST Biometrics Secure Access Solution, as Part of Multi-million Dollar Expansion 2Crossmatch Fingerprint Readers Help Retailer Prevent Data Breaches at Point-of-Sale 2
... Institute (NHGRI), one of the National Institutes of Health ... will target 13 more organisms as part of its ... biological knowledge and improve human health. , The National ... a federally chartered committee that advises NHGRI on program ...
... Ever wondered why you aren't able to swat a fly? ... in its decision to jump rather than to fly out ... of interest to those building autonomously navigating robots", according to ... be presenting her work on triggered escape response at the ...
... first time, scientists have identified an ant species that ... , Stanford University biologist Deborah M. Gordon and ... issue of the journal Nature. The discovery was made ... student Megan E. Frederickson in the Amazon jungle of ...
Cached Biology News:NHGRI Selects 13 More Organisms for Genome Sequencing 2NHGRI Selects 13 More Organisms for Genome Sequencing 3NHGRI Selects 13 More Organisms for Genome Sequencing 4NHGRI Selects 13 More Organisms for Genome Sequencing 5Ants, not evil spirits, create devil's gardens in the Amazon rainforest, study finds 2Ants, not evil spirits, create devil's gardens in the Amazon rainforest, study finds 3Ants, not evil spirits, create devil's gardens in the Amazon rainforest, study finds 4
(Date:4/30/2015)... (PRWEB) April 30, 2015 SureClinical, a ... Julie I. Morris has joined SureClinical’s executive team as ... experienced biotech and technology executive with over 25 years ... 500 companies. , Ms. Morris joins SureClinical from ... company. She was CFO of MBI from its founding ...
(Date:4/30/2015)... 30, 2015 Sterlitech is proud ... the PX, PY, and PZ Polyacrylonotrile (PAN) ... disks. Designed to be ideal for oil ... can fill critical process requirements where the presence ... , “New regulations on industrial wastewater discharge and ...
(Date:4/29/2015)... April 29, 2015 SPIE DSS, ... imaging on the East Coast, reaffirmed its value last ... applications developers, and industry suppliers to meet and advance ... multiple applications. , The event included 55 conferences in ... scientific sensing and imaging -- and an exhibition showcasing ...
(Date:4/29/2015)... April 29, 2015   Doximity , the leading ... US doctors as members, announced a new, instant method ... Using Doximity." With one click, all US physicians can ... vitae (CV) for an open job opportunity, and a ... Eighty seven percent of physicians are open to ...
Breaking Biology Technology:Julie I. Morris Joins SureClinical as Chief Financial Officer 2Sterlitech Offers New Ultrafiltration Membranes 2SPIE DSS provides venue for collaboration vital to research, applications advances 2SPIE DSS provides venue for collaboration vital to research, applications advances 3SPIE DSS provides venue for collaboration vital to research, applications advances 4Doximity Offering Hospitals Easy, Efficient Physician Application Tool 2
... Trojans hauled that giant wooden statue inside their walls, ... in the streets, people have been examining gift horses ... - within reason. , ,The state Building Commission will ... landmark plan to build the first phase of the ...
... come as a surprise to know that I'm often asked to ... course, a few basic rules of thumb that guide any product ... , Be passionate about the product. , Avoid hokey scenarios. , ... Practice, practice, practice. , ,These are the top-line criteria for a ...
... announced Wednesday that it has begun shipping Gen2 RFID-tagged cases ... Auto-ID research lab in Neenah, Wis., to thank for it. ... of Huggies brand diapers is the company's next step in ... extensive research and testing on the compatibility of Gen2 hardware ...
Cached Biology Technology:Seven good reasons to approve the Institutes for Discovery 2Seven good reasons to approve the Institutes for Discovery 3Secrets of a great demo 2Secrets of a great demo 3Neenah lab helps firm ship RFID-tagged products to Wal-Mart 2
Extra Large bottle for hybridization 300 x 70mm...
Request Info...
... The Thermo IEC Microtube Rotor is ... have to change rotors. You can ... and PCR tubes. Exclusive polypropylene rotor design ... repeatedly autoclaved. Polypropylene rotors are fully ...
... Wellpro 384, compatible with ... liquid handling system for ... and accurately prepares large ... The Wellpro also ...
Biology Products: