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Children with rare, incurable brain disease improve after genetherapy by ferujkll sdff
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Children with rare, incurable brain disease improve after genetherapy |
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The first-in-humans achievement may also be helpful for more commondiseases such as Parkinson's that involve nerve cell damage causedby lack of a crucial molecule in brain tissue. The results arereported today in the journal Science Translational Medicine. The children in the study, who ranged in age from 4 to 6, inheriteda rare disease known as aromatic L-amino acid decarboxylasedeficiency, or AADC. Patients with AADC are born without an enzymethat enables the brain to produce the neurotransmitter dopamine.They generally die in early childhood. In a phase 1 clinical trial led by Dr.
Wuh-Liang Hwu, of theNational Taiwan University Hospital, surgeons used a deliveryvehicle called an adeno-associated virus type 2 vector to transportthe AADC gene into localized areas of the brains of three girls anda boy. Before therapy, the children showed practically no spontaneousmovement and their upper eyelids continually drooped. Afterreceiving the corrective gene, the children gradually gained somehead movement. Sixteen months afterward, the children's weight hadincreased, one patient was able to stand and the other three wereable to sit up without support.
The study shows gene therapy that targets AADC deficiency iswell-tolerated and leads to improved motor development andfunction, according to co-authors Dr. Barry Byrne, director of UF'sPowell Gene Therapy Center, and Richard O. Snyder, director of UF'sCenter of Excellence for Regenerative Health Biotechnology. Bothare members of the UF Genetics Institute. "The children in this study have the most severe form ofinherited movement disorder known, and the only treatments so farhave been supportive ones," said Byrne, a pediatriccardiologist and associate chairman of the department of pediatricsin the College of Medicine.
"It is gratifying to see it ispossible to do something to help them, other than providing feedingtubes and keeping them safe. This absolutely opens the door to thepossibility of even earlier treatment of neurological diseases bydirect gene transfer, and has implications for Parkinson's disease,ALS and even cognitive diseases such as dementia when caused bygene defects." The Powell Gene Therapy Center provided expertise to the Taiwanesephysicians on treating the patients and engineering the correctivegene that spurs production of the absent AADC enzyme. UF's Centerof Excellence for Regenerative Health Biotechnology manufacturedthe vector, packaging genetic material it received from Taiwan intovirus particles that were purified, characterized and tested forsterility and stability before being shipped to the clinic for usein patients. "We are ecstatic that we manufactured a product that providedtherapeutic benefit to these patients," said Snyder, anassociate professor in UF's department of molecular genetics andmicrobiology. "What really makes it special is there are justa handful of examples of gene therapy in children in the world, andthese patients all improved." Doctors injected the AADC vector into a brain area called theputamen, a site known for AADC activity and part of a"loop" of brain connections related to movement.
Postoperative CT and MRI scans of the patients showed no evidenceof bleeding and all four patients were discharged within a week.Three to six months after gene transfer, all the children hadgained weight, including one patient who doubled her weight withina year. Before gene therapy, all patients showed low raw scores incognition and motor development on a scale called the ComprehensiveDevelopmental Inventory for Infants and Toddlers. Afterward, scoresin both areas increased. Parents reported the children also slept better and had improvedeye coordination, emotional stability and body temperaturestability.
Eight additional children -- four in Taiwan and four in the UnitedStates -- are expected to receive the experimental treatment, Byrnesaid. I am CPUs writer, reports some information about gas scooter reviews , moped free shipping.
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