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

nd in many patients with familial ALS, which accounts for about 2 percent of all cases.

SOD1 provides instructions for making an enzyme called superoxide dismutase, which is found throughout the body and breaks down toxic molecules that can be damaging to cells. When mutated, the SOD1 gene yields a faulty version of the enzyme that is especially harmful to motor neurons. One of the mutations, which is found in about half of all familial ALS patients, is particularly devastating, with death usually coming within 18 months of diagnosis. SOD1 has also been implicated in other types of ALS, called sporadic ALS, which means the therapy could prove beneficial for larger numbers of patients suffering with this disease.

Earlier work by Dr. Kaspar and others found that they could halt production of the mutated enzyme by blocking SOD1 expression, which in turn, they suspected, would slow ALS progression. To test this hypothesis, the researchers would not only need to come up with an approach that would block the gene, but also figure out how to specifically target cells implicated in the disease, which include motor neurons and glial cells. What's more, the therapy would preferably be administered noninvasively instead of direct delivery via burr holes drilled into the skull.

Dr. Kaspar's team accomplished the second part of this challenge in 2009, when they discovered that adeno-associated virus serotype 9 (AAV9) could cross the blood-brain barrier, making it an ideal transport system for delivering genes and RNA interference strategies designed to treat disease.

In this new work, funded by the National Institutes of Health, the researchers blocked human SOD1, using a technology known as short hairpin RNA, or shRNA. These single strands of RNA are designed in the lab to seek out specific sequences found in the human SOD1 gene, latch onto them and block gene expression.

In one of the mouse models used in the study, ALS develops
'/>"/>

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:7/9/2014)... on the verge of an economy driven by methane, the ... and is undergoing a production boom. It has poised the ... is casting serious doubts over just how climate-friendly it is, ... (C&EN), the weekly news magazine of the American Chemical ... at C&EN, explains that when burned as a fuel to ...
(Date:7/9/2014)... Can thyroid hormone protect neuronal function and increase ... Ailing Fu and her team, School of Pharmaceutical ... in which aged mice were administered with low ... showed that the aged rats exhibited an obvious ... rate from 60% to 93%. The underlying mechanism ...
(Date:7/9/2014)... They are amongst the most numerous inhabitants of the ... . Not visible to the naked eye, when they ... sized patches, they are even visible on satellite images. ... responsible for approximately half of the global photosynthesis output," ... University Jena (Germany). In the process the greenhouse gas ...
Breaking Biology News(10 mins):Short circuit in the food web 2
... of scientists, including University of Colorado School of Medicine ... disease related to an inability to process Vitamin B12. ... "Some people with rare inherited conditions cannot process vitamin ... a geneticist and senior author of a paper about ...
... key role in preserving biological diversity. A new study carried ... (SLU), among others, indicates that if the world,s religious leaders ... positioned to do so. Our study investigates how ... they overlap areas that are important for global biological diversity, ...
... $4.5 million over the next five years for a ... detecting disorders and conditions can and should become a ... one of four projects launched today by the National ... providing genetic sequencing as part of, or instead of, ...
Cached Biology News:Peering into genetic defects, CU scientists discover a new metabolic disease 2Peering into genetic defects, CU scientists discover a new metabolic disease 3Religious leaders can be key to biological diversity 2UCSF receives $4.5M to study value of gene sequencing in newborns 2UCSF receives $4.5M to study value of gene sequencing in newborns 3UCSF receives $4.5M to study value of gene sequencing in newborns 4
(Date:1/15/2014)... , Jan. 15, 2014 TaiGen Biotechnology Company, Limited ... agreement with R-Pharm, a leading Russian pharmaceutical company, to ... Russian Federation , Turkey ... States (CIS). Nemonoxacin is a novel antibiotic for the treatment ...
(Date:1/14/2014)... CA (PRWEB) January 14, 2014 Histogen, ... the products of cells grown under simulated embryonic conditions, ... license agreement with Suneva Medical, Inc. for physician-dispensed aesthetic ... , This agreement is an amendment to ...
(Date:1/14/2014)... 14, 2014 EquitiesIQ, a leading informational ... ALQA). Alliqua is an emerging biomedical company acquiring, developing, ... care market. , Free report download: http://equitiesiq.com/reports/alliqua/ ... seasoned management team and Board, which launched the company’s ...
(Date:1/14/2014)... 14, 2014 iLab Solutions, the leader in ... new Director of Product Strategy. In this role, Michelle will ... sub-teams to guide in the development of iLab products. Her ... maximum possible benefit to the scientific community by offering the ...
Breaking Biology Technology:TaiGen Biotechnology Signed Exclusive License Agreement with R-Pharm for Nemonoxacin (Taigexyn(R)) 2TaiGen Biotechnology Signed Exclusive License Agreement with R-Pharm for Nemonoxacin (Taigexyn(R)) 3TaiGen Biotechnology Signed Exclusive License Agreement with R-Pharm for Nemonoxacin (Taigexyn(R)) 4Histogen and Suneva Medical Expand License for Cell Conditioned Media-based Aesthetic Products Internationally 2Histogen and Suneva Medical Expand License for Cell Conditioned Media-based Aesthetic Products Internationally 3EquitiesIQ Initiates Coverage of Alliqua, Inc. 2EquitiesIQ Initiates Coverage of Alliqua, Inc. 3iLab Solutions Announces Michelle Detwiler as the New Director of Product Strategy 2
... Inc., a developer of innovative therapeutics for diseases ... results for the company,s lead product candidate, OTO-104, ... demonstrated that a single intratympanic (IT) injection of ... corticosteroid dexamethasone, provided significant protection against both noise-induced ...
... Feb. 22, 2011 Jennerex, Inc. (San ... appointment of James M. Burke, M.D. to the newly-created ... be responsible for all global clinical research activities related ... has been working with Jennerex in an advisory capacity ...
... KONG, Feb. 22, 2011 /PRNewswire-Asia-FirstCall/ -- China Cord Blood Corporation ... first and largest cord blood banking operator in China, today ... was held on February 18, 2011, in Hong Kong. ... Albert Chen and Mark Chen to serve on the Board ...
Cached Biology Technology:Otonomy's OTO-104 Demonstrates Hearing Loss Protection and Hearing Recovery in Preclinical Studies 2Otonomy's OTO-104 Demonstrates Hearing Loss Protection and Hearing Recovery in Preclinical Studies 3Jennerex Appoints James M. Burke, M.D. Vice President, Clinical Research 2China Cord Blood Corporation Announces Results of 2010 Annual General Meeting 2
... is a new product number, created ... If showing no availability yet, please ... (E6152) or contact customer service for ... diam. 11.25 6.40 in. ...
... Analysis of large numbers of samples may ... which requires minimum operator attention. System control ... and interface CE 4900. The elegant, powerful ... Windows XP and Windows 98 and 2000., The ...
Mouse monoclonal antibody to QPCT - glutaminyl-peptide cyclotransferase (glutaminyl cyclase)...
... number is a new ... easily match Cornings product ... availability yet, please order ... number (Z35,347-7) or contact ...
Biology Products: