Spectrum: Autism Research News
In pursuit of gene therapies for autism-linked conditions
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
Featured Articles
A quest for Quincy: Gene therapies come of age for some forms of autism
A gene therapy for Angelman syndrome stands at the forefront of efforts to treat autism-linked conditions that stem from single genes.
Lessons from n-of-1 trials: A conversation with Joseph Gleeson
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.
What next for Angelman?
A meeting in Texas reckons with the future of treatment, following two setbacks in 2020.
RNA therapy restores gene function in monkeys modeling Angelman syndrome
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.
Latest ‘prime-editing’ tools tackle delivery, safety issues
The gene-editing advances make it easier to target specific tissues in mice and detect off-target effects.
Angelman therapy appears safer in restarted trial
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.
Alternative gene-therapy approaches take aim at Rett syndrome
Methods that selectively increase levels of the Rett protein make for safer and more effective treatment strategies, some researchers say.
Neuron-specific virus overcomes barriers to brain-related gene therapy
A new viral variant can deliver genes exclusively to the brain, overcoming a key hurdle in treating neurological conditions using gene therapy.
New gene therapy methods deliver promise
Two unpublished studies detail improved techniques for delivering gene therapies to the brain.