Spectrum: Autism Research News
In October 2020, freelance journalists Lydia Denworth and Brendan Borrell traced one woman’s journey to devise a gene therapy for Angelman syndrome, which affects her daughter, Quincy. Here, we revisit this and other Spectrum articles covering experimental gene therapies for autism-linked genetic conditions — and some new methods that may help scientists on their quests.
Cite this article: https://doi.org/10.53053/QDSJ8213
A gene therapy for Angelman syndrome stands at the forefront of efforts to treat autism-linked conditions that stem from single genes.
Some conditions are too rare for conventional drug trials, leading some scientists to test bespoke treatments in single participants. Gleeson discusses the merits — and limitations — of these tiny trials.
A meeting in Texas reckons with the future of treatment, following two setbacks in 2020.
The result raises hopes for an ongoing clinical trial in people — and offers fresh insight into the biology of imprinting and the UBE3A antisense transcript.
The gene-editing advances make it easier to target specific tissues in mice and detect off-target effects.
Interim results from the previously paused trial suggest that doses of the experimental gene therapy drug GTX-102 are well tolerated in children with the autism-linked condition.
Methods that selectively increase levels of the Rett protein make for safer and more effective treatment strategies, some researchers say.
A new viral variant can deliver genes exclusively to the brain, overcoming a key hurdle in treating neurological conditions using gene therapy.
Two unpublished studies detail improved techniques for delivering gene therapies to the brain.