Navigation Links
Gene therapy cures a severe paediatric neurodegenerative disease in animal models
Date:7/2/2013

Sanfilippo Syndrome type A, or Mucopolysaccharidosis type IIIA (MPSIIIA), is a neurodegenerative disease caused by mutations in the gene that encodes the enzyme sulfamidase. Mutations in this gene lead to deficiencies in the production of the enzyme, which is essential for the breakdown of substances known as glycosaminoglicans. If these substances are not broken down, they accumulate in the cells and cause neuroinflammation and organ dysfunction, mainly in the brain, but also in other parts of the body. Children born with this mutation are diagnosed from the age of 4 or 5. They suffer neurodegeneration, causing mental retardation, aggressiveness, hyperactivity, sleep alterations, loss of speech and motor coordination, and they die in adolescence.

A team of researchers headed by the director of the UAB's Centre for Animal Biotechnology and Gene Therapy (CBATEG), Ftima Bosch, has developed a gene therapy treatment that cures this disease in animal models, with pre-clinical studies in mice and dogs. The treatment consists of a single surgical intervention in which an adenoassociated viral vector is injected into the cerebrospinal fluid, the liquid that surrounds the brain and the spinal cord. The virus, which is completely harmless, genetically modifies the cells of the brain and the spinal cord so that they produce sulfamidase, and then spreads to other parts of the body, like the liver, where it continues to induce production of the enzyme.

Once the enzyme's activity is restored, glycosaminoglican levels return to normal for life, their accumulation in cells disappears, along with the neuroinflammation and dysfunctions of the brain and other affected organs, and the animal's behaviour and its life expectancy return to normal. While mice with the disease lived only up to 14 months, those given the treatment survived as long as healthy ones.

This is a joint project between the UAB and the pharmaceutical company Esteve. The study has
'/>"/>

Contact: Fàtima Bosch
Fatima.bosch@uab.cat
34-935-814-182
Universitat Autonoma de Barcelona
Source:Eurekalert

Page: 1 2

Related medicine news :

1. Pufferfish neurotoxin may serve as treatment for cancer therapy related pain
2. A circuitous route to therapy resistance
3. Vein Therapy News Magazine Publishes Laser Facelift Article by Dr. Robin Fleck
4. Rural women less likely to get radiation therapy after lumpectomy for breast cancer
5. New understanding of why anti-cancer therapy stops working at a specific stage
6. Past Life Regression Therapy: Valid Treatment or Ridiculous Foo Foo; Researcher and Author Dr. Heather Friedman Rivera on Dr. Carol Francis Radio Talk Show
7. Genetics of cervical cancer raise concern about antiviral therapy in some cases
8. PREFERRED Therapy Providers, Inc. and WellCare of Arizona Finalize Their Agreement to Offer Medicare Advantage Plan to PREFERRED's Arizona Provider Network
9. June's Release of CARD's "Mission Possible: Portraits of Hope" Features Southern California Child and His Remarkable Progress with Intensive Autism Therapy
10. Filtersfast.com Launches Spazazz Aromatherapy Spa & Bath Crystals
11. Newly Released Continuing Education Courses Provide Physical and Occupational Therapy Professionals With Opportunities To Expand Skill Sets
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/25/2014)... December 25, 2014 The short film “Color ... created in honor of a true 9/11 hero, Welles Remy ... festivals including the 24 hours of Nuremberg International Short Film ... , Actress, screenwriter and director Luciana Lagana ... film together with her husband, Gregory Graham - ...
(Date:12/25/2014)... December 25, 2014 Recently, LunaDress.co.uk has updated its ... and related accessories. Now, all of the models at LunaDress.co.uk come ... a groom lifts the wedding veil can turn out to be ... charm. A wedding veil can always bring surprise to a wedding. ... on how to choose veils for a big day . ...
(Date:12/25/2014)... Dennis Thompson HealthDay ... Researchers could be closing in on a "fountain of youth" ... the health of older adults, a new study suggests. ... given a drug that targets a genetic signaling pathway linked ... Novartis report. The experimental medication, a version ...
(Date:12/25/2014)... Residents at Abernethy Laurels ... their generous spirit by providing holiday gifts to ... Network- Grandparents Raising Grandchildren program. , Following a ... largest Christmas tree on the campus of the ... of the Abernethy Village Association (AVA). Each holiday ...
(Date:12/24/2014)... Epigenetics finds applications in a large number of ... developmental biology, and research for metabolic and genetic disorders. ... widely used techniques for the discovery and development of ... is rapidly growing worldwide. In 2012, there were 14.1 ... 32.6 million people living with cancer (within five years ...
Breaking Medicine News(10 mins):Health News:The True 9/11 Hero Story “Color of Honor” Premieres in Germany and Bangladesh: Luciana Lagana Plays the Mother of the Lead and Co-Produces with Blaise Dolcemaschio. 2Health News:The True 9/11 Hero Story “Color of Honor” Premieres in Germany and Bangladesh: Luciana Lagana Plays the Mother of the Lead and Co-Produces with Blaise Dolcemaschio. 3Health News:LunaDress Provides Professional Tips On Shopping Wedding And Prom Dresses 2Health News:Researchers Take 'First Baby Step' Toward Anti-Aging Drug 2Health News:Researchers Take 'First Baby Step' Toward Anti-Aging Drug 3Health News:Retirement Community Residents Make Impact During Holiday Season 2Health News:Global Epigenetics Market Was Valued at $413.24 Million in 2014 - New Research Report by MarketsandMarkets 2Health News:Global Epigenetics Market Was Valued at $413.24 Million in 2014 - New Research Report by MarketsandMarkets 3
... are more common in women than in men, says Coping ... School. Scientists are// examining hormonal and biological factors, as well ... ,Women are twice as likely to suffer from ... are three times as likely to have agoraphobia (fear of ...
... by retina surgeons at The Methodist Hospital shows unprecedented ... treated with Lucentis? for wet// age-related macular degeneration (AMD), ... Journal of Medicine. AMD is a major cause of ... causes of blindness in people over 60. ...
... toll from the mosquito-borne virus has risen to six in ... died of the disease in the paediatric ward of King ... the Farrukhabad district hospital,' V.S. Nigam, health department spokesman told ... cases has shot up to 199 in the state with ...
... that behavioral therapies can benefit children who have problems falling ... ,The American Academy of Sleep Medicine (AASM) appointed a team ... and toddlers. ,According to study author Jodi A. ... shown 20 to 30 percent of young children have sleep ...
... Pradesh villager suffering from a serious kidney ailment died ... premier hospital here//, apparently because he could not afford ... of a window of the nephrology ward of the ... he was being treated, hospital sources told IANS. ...
... debate on the issue of organ donation. The author advocates ... will help in meeting the excessive demand for transplants and ... is an organ transplant surgeon by profession has opined that ... requirement for organs whilst remaining on the right side of ...
Cached Medicine News:Health News:Results for Wet Age-Related Macular Degeneration Show Significant Vision Improvements 2
(Date:12/24/2014)... , Dec. 23, 2014 PuraMed BioScience®, Inc., ... over-the-counter (OTC) medicinal and healthcare products, announced it received ... H, a hemp-based, advanced headache relief product, for planned ... Colorado , Washington State ... With the MigraPure H Advanced headache relief gel ...
(Date:12/24/2014)...  Advanced Medical Isotope Corporation ("AMIC") (OTCQB: ADMD), a ... development of brachytherapy devices and medical isotopes for ... has filed a de novo submission ... marketing clearance for its patented Y-90 RadioGel(TM) device ... Drug and Cosmetic Act (the "Act").  ...
(Date:12/24/2014)... 24, 2014 LifeScienceIndustryResearch.com ... Disposable Syringe Industry of 178 pages providing ... intelligence library. The 2014 Market ... is a professional and deep research report ... analysis, the report introduces Disposable Syringe basic ...
Breaking Medicine Technology:PuraMed BioScience Nears Final Stages Needed for Distribution of MigraPure H 2PuraMed BioScience Nears Final Stages Needed for Distribution of MigraPure H 3Advanced Medical Isotope Corporation Files a "de novo" Submission for Y-90 RadioGel(TM) Device 2Advanced Medical Isotope Corporation Files a "de novo" Submission for Y-90 RadioGel(TM) Device 3World Disposable Syringe Market 2014 Report: China and Regional Analysis 2World Disposable Syringe Market 2014 Report: China and Regional Analysis 3World Disposable Syringe Market 2014 Report: China and Regional Analysis 4World Disposable Syringe Market 2014 Report: China and Regional Analysis 5
... Group (MRG), the global authority on medical technology ... research data for multiple geographies, including the US, ... MRG,s peripheral vascular reports cover minimally invasive ... stenting, percutaneous transluminal balloon angioplasty, and synthetic surgical ...
... RO, ROG; OTCQX: RHHBY) and study investigators from Harvard ... the PROTECT ( Pr o-BNP O utpatient ... T herapy) trial, a strategy of NT-proBNP guided heart ... in total cardiovascular events, the primary composite endpoint of ...
Cached Medicine Technology:New Peripheral Vascular Device Market Research Data Covering Multiple Geographies Released by Millennium Research Group 2NT-proBNP Guided Therapy Keeps Heart Failure Patients out of the Hospital Through a Reduction in Cardiovascular Events 2NT-proBNP Guided Therapy Keeps Heart Failure Patients out of the Hospital Through a Reduction in Cardiovascular Events 3
Diamond Point Bone Probe with Ortho-Grip® silicone rubber palm style handles and,stainless steel impactor caps....
Steroid-eluting, single-pass lead. CapSure VDD2 optimizes VDD therapy and offers easier handling, proven performance, and choice....
CapSureSP Novus 5594 is a J-shaped, endocardial, tined pacing lead. provide the stable, low pacing thresholds and large sensing potentials already proven with CapSure SP steroid-eluting leads but wi...
Steroid eluting pacemaker leads...
Medicine Products: