A disease which prevents children from walking or talking has apparently been cured by a pioneering gene therapy to correct genetic errors in their DNA.
The three patients in the study all sufferer from Wiskott-Aldrich syndrome, a disease which sees children’s development reverse from age 1-2 as part of their brain gets destroyed.
Gene treatment enabled patients to go to school
However, after being injected with the genetically modified virus developed by researchers at Milan’s San Raffaele Scientific Institute, all three were able to not only walk and talk but even go to school.
Study lead Dr Alessandra Biffi said: “The outcome has been very positive, they’re all in very good condition, with a normal life and going to kindergarten at an age when their siblings were unable to talk. It is something which is very pleasing to us.”
However, gene therapy is a field that has been hampered by serious concerns about safety and Dr Biffi warned that all treatments have side effects and these patients needed to be followed for longer.
SWAN lottery grants
The three children were picked for treatment from families with a history of metachromatic leukodystrophy, but before their brain function started to decline.
Further hope may also been offered to support the families of disabled children with undiagnosed genetic condition as SWAN UK recently became one of seven projects to make it through to the finals in the Best Health project category at the National Lottery Awards.
Supporting Families with Children with Undiagnosed Genetic Conditions (SWAN) offers life-changing support to the families of thousands of children with learning disabilities who have an unexplained genetic condition. Without a diagnosis, parents can be left without answers to soul searching questions and feel like their children don’t ‘fit in’. Many worry about having more children and the same thing happening again.
Experts in undiagnosed genetic conditions
SWAN UK has received more than £300,000 of National Lottery funding to provide information and support to families. This has helped to develop a network of professionals with expertise in undiagnosed genetic conditions. This expertise is shared via social media, its dedicated website, a series of leaflets and social events arranged by SWAN UK for families.
The winning project in each category will receive national recognition on a star-studded BBC One TV show in early September and, in addition to the Lottery funding they have already received, will receive a £2,000 cash prize to spend on their project.
