Navigation Links
Gene Therapy Shows Promise for Rare Children's Brain Disorder
Date:12/19/2012

urgh.

The improvements in children's actual functioning were small, Escolar noted. And one problem, she said, is that there are no good "natural history" studies on kids with Canavan disease. That is, researchers don't fully understand the variance in how children with the disease naturally fare.

It's hard to know how much of the symptom improvement, and overall survival, was due to the gene therapy itself, Escolar said. Advances in how doctors care for kids' complications, such as pneumonia, might have affected survival rates.

Still, Escolar said, "the safety findings, alone, are a huge step forward."

Since this trial began, scientists have also made advances in the viral vectors that could be used for gene therapy. Better ones, that may not need to be directly infused into the brain, are under study in animals -- and close to being studied in humans, Escolar noted.

"Now this study needs to be repeated using better vectors, along with better natural history data," Escolar said.

Gene therapy is not the only approach to Canavan under study, said Morris Baslow, a scientist at the Nathan S. Kline Institute for Psychiatric Research in Orangeburg, N.Y.

Other research groups, he said, are trying to better understand how the chemical NAA works in the brain, and hopefully come up with other approaches to treat Canavan disease, including medications.

When it comes to gene therapy, Baslow agreed that it should start very early, soon after birth. He also said the "ideal" method would be to use a viral vector that specifically targets brain cells called oligodendrocytes -- which are responsible for making the protective sheaths around nerves.

Leone agreed that that type of vector would be better. But it doesn't exist yet.

Ultimately, a combination of therapies may be needed to best treat, or even cure, Canavan disease. "Our feeling is, in the future, gene therapy in association with medication that targets the metaboli
'/>"/>

Copyright©2012 ScoutNews,LLC.
All rights reserved  

Page: 1 2 3 4

Related medicine news :

1. People with HIV hospitalized less often since combination antiretroviral drug therapy introduced
2. Shaft Medical San Diego now offers micro needle sclerotherapy to treat scary “spider” veins.
3. Churchview Supportive Living to Host Music Therapy Informational Program
4. UAlberta medical researchers discover new potential chemotherapy
5. New hormone therapy shows promise for menopausal symptoms in animal model
6. Older and younger chronic leukemia patients may need different therapy
7. Novel test identifies patients most likely to benefit from ALK inhibition therapy
8. Combo Therapy May Help Ease Sleep Apnea at High Altitude
9. Potential gene therapy approach to sickle cell disease highlighted at American Society of Hematology
10. Dr. Farshchian: Labrum Tear is Now Being Treated With Cell Therapy at the Center for Regenerative Medicine Using
11. Moffitt researchers say effective immunotherapy for melanoma hinges on blocking suppressive factors
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Gene Therapy Shows Promise for Rare Children's Brain Disorder
(Date:8/21/2014)... is to focus on her body in terms of ... her as a complete human being with thoughts and ... in the media, how does it affect individual romantic ... Quarterly , a SAGE journal, finds that more objectification ... incidents of sexual pressure and coercion., Researchers Laura R. ...
(Date:8/21/2014)... (PRWEB) August 21, 2014 The results ... today in The Journal of the American Medical Association ... invasive cancers and reduces unnecessary recalls. Memorial Healthcare System ... the study. , “This study confirms what we already ... harmful cancers we want to find and, at the ...
(Date:8/21/2014)... The report "Industrial Flue Gas Treatment Systems ... DeNOx, Particulate Control, Mercury Control) by Application (Power ... - Global Trends & Forecasts to 2019" defines ... systems & services market with analysis and projection ... value. The industrial flue gas treatment systems & ...
(Date:8/21/2014)... 21, 2014 Lauren Collura, a mother of ... “Back to the Boardwalk” Facebook sweepstakes. Lauren, her husband, and ... four-bedroom beachfront house in Lavallette beginning this Saturday. , ... Boardwalk” sweepstakes. Last year, Horizon Blue Cross Blue Shield of ... Superstorm Sandy and decided to make it a new summer ...
(Date:8/21/2014)... 2014 American Addiction Centers, a leading ... individuals with drug and/or alcohol addiction, has been named ... Private Companies. , The company is ranked 7th on ... and 4th on the list in the “Nashville Metro ... 995%, American Addiction Centers (AAC) ascended to number 474 ...
Breaking Medicine News(10 mins):Health News:Objectification in romantic relationships related to sexual pressure and coercion 2Health News:Dr. Mary Hayes: Study Shows 3D Mammography Finds More Invasive Cancers While Reducing Unnecessary Recalls 2Health News:Industrial Flue Gas Treatment Systems Market Estimated to Worth $59, 546.7 Million by 2019 – Report by MarketsandMarkets 2Health News:Industrial Flue Gas Treatment Systems Market Estimated to Worth $59, 546.7 Million by 2019 – Report by MarketsandMarkets 3Health News:Industrial Flue Gas Treatment Systems Market Estimated to Worth $59, 546.7 Million by 2019 – Report by MarketsandMarkets 4Health News:American Addiction Centers Named to Inc. 500 List Recognition for Three-Year Sales Growth of 995% 2
... adults in their late 30,s received a flu shot during ... study released today says. But about 65 percent were ... percent said they were following the issue very or moderately ... adults during the 2009-2010 H1N1 influenza epidemic, this second of ...
... Doheny HealthDay Reporter , TUESDAY, Jan. 24 (HealthDay ... the risk of getting ovarian cancer, but new research shows ... surviving the deadly disease. Women with invasive epithelial ovarian ... women without the genetic variations, according to an analysis of ...
... practiced as part of routine care for people with ... pressure and cholesterol levels and keep them under control, ... February issue of Diabetes Care . Researchers ... cohort study of more than 30,000 people with diabetes ...
... , TUESDAY, Jan. 24 (HealthDay News) -- Psychedelic ... suggest two small brain imaging studies that seem to ... mushrooms -- affects the brain. One study included ... blood while magnetic resonance imaging (MRI) scanners measured changes ...
... HealthDay Reporter , TUESDAY, Jan. 24 (HealthDay ... and other signs of gastroesophageal reflux don,t get additional ... And, taking these medications when there are ... a respiratory infection, reports the study. "There,s a ...
... , TUESDAY, Jan. 24 (HealthDay News) -- Obstetricians ... partner violence, including during prenatal visits, according to ... and Gynecologists. In addition, ob-gyns should offer ... on hand to give to patients who are ...
Cached Medicine News:Health News:Young adults responded well to swine flu 2Health News:Gene Mutations May Boost Ovarian Cancer Survival: Study 2Health News:Gene Mutations May Boost Ovarian Cancer Survival: Study 3Health News:Lifestyle counseling reduces time to reach treatment goals for people with diabetes 2Health News:Could 'Magic' Mushrooms Ease Depression? 2Health News:Could 'Magic' Mushrooms Ease Depression? 3Health News:Heartburn Meds Won't Help, May Harm Kids With Asthma 2Health News:Heartburn Meds Won't Help, May Harm Kids With Asthma 3Health News:Ob-Gyns Should Screen for Domestic Abuse: Experts 2
(Date:8/21/2014)... Va. , Aug. 21, 2014 ... quality and Manufacturing Intelligence , today announces ... Conference. The event will be held Oct. 9-10, ... and is designed to showcase the necessity for ... of metrics and Manufacturing Intelligence to gain insight ...
(Date:8/21/2014)... Aug. 21, 2014  The board of directors of ... provider of clinical trial imaging solutions, today announced that ... financial officer, effective immediately.  Mr. Groff has held the ... He joined VirtualScopics in January 2006 as an accounting ... "When the board of VirtualScopics asked Jim ...
(Date:8/21/2014)... 21, 2014  Cyberonics, Inc. (NASDAQ: CYBX ) ... Quarterly highlights 1   Operating ... the first quarter of fiscal 2014, and other achievements, include: ... increase of 6.8%; , Strong international net sales of ... basis; , U.S. net product sales reached a new ...
Breaking Medicine Technology:InfinityQS to Host Manufacturing Intelligence Conference in Chicago 2VirtualScopics, Inc. Names James Groff Chief Financial Officer 2VirtualScopics, Inc. Names James Groff Chief Financial Officer 3VirtualScopics, Inc. Names James Groff Chief Financial Officer 4Cyberonics Reports Fiscal 2015 First Quarter Results 2Cyberonics Reports Fiscal 2015 First Quarter Results 3Cyberonics Reports Fiscal 2015 First Quarter Results 4Cyberonics Reports Fiscal 2015 First Quarter Results 5Cyberonics Reports Fiscal 2015 First Quarter Results 6Cyberonics Reports Fiscal 2015 First Quarter Results 7Cyberonics Reports Fiscal 2015 First Quarter Results 8Cyberonics Reports Fiscal 2015 First Quarter Results 9Cyberonics Reports Fiscal 2015 First Quarter Results 10Cyberonics Reports Fiscal 2015 First Quarter Results 11Cyberonics Reports Fiscal 2015 First Quarter Results 12Cyberonics Reports Fiscal 2015 First Quarter Results 13Cyberonics Reports Fiscal 2015 First Quarter Results 14Cyberonics Reports Fiscal 2015 First Quarter Results 15Cyberonics Reports Fiscal 2015 First Quarter Results 16
... , STAMFORD, Conn., Nov. 13 ... will be releasing a free trial version of its Zargis ... conference, taking place this November 15-17 in Orlando. Zargis is ... the companion hardware -- the 3M Littmann Electronic Stethoscope Model ...
... BERWYN, Pa., Nov. 13 /PRNewswire-FirstCall/ -- Encorium Group, ... multinational clinical research organization (CRO) conducting studies in over ... and biotechnology companies, today announced that it has appointed ... 7, 2009. Ms. Laitinen, age 43, will replace ...
Cached Medicine Technology:Zargis to Release Trial Version of Cardioscan while Exhibiting with 3M(TM) Littmann(R) at American Heart Association Conference 2Zargis to Release Trial Version of Cardioscan while Exhibiting with 3M(TM) Littmann(R) at American Heart Association Conference 3Encorium Group, Inc. Appoints Sari Laitinen to Board of Directors 2Encorium Group, Inc. Appoints Sari Laitinen to Board of Directors 3
... Laboratories is a national reference laboratory and ... and development. ARUP offers an extensive test ... tests in clinical and anatomic pathology. Owned ... clients include more than half of the ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... ARUP Laboratories is a national reference ... laboratory research and development. ARUP offers an ... unique medical tests in clinical and anatomic ... ARUP Laboratories' clients include more than half ...
Medicine Products: