In pursuit of gene therapies for autism-linked conditions
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.
Latest ‘prime-editing’ tools tackle delivery, safety issues
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.
RNA therapy restores gene function in monkeys modeling Angelman syndrome
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.
Lessons from n-of-1 trials: A conversation with Joseph Gleeson
What next for Angelman?
A meeting in Texas reckons with the future of treatment, following two setbacks in 2020.
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.
Angelman therapy appears safer in restarted trial
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.
Neuron-specific virus overcomes barriers to brain-related gene therapy
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.
Alternative gene-therapy approaches take aim at Rett syndrome
New gene therapy methods deliver promise
Two unpublished studies detail improved techniques for delivering gene therapies to the brain.
New gene therapy methods deliver promise
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.
A quest for Quincy: Gene therapies come of age for some forms of autism
Explore more from The Transmitter
Rat neurons thrive in a mouse brain world, testing ‘nature versus nurture’
Neurons from the two rodents can wire up together to form functional circuits—all while maintaining some species-specific properties, two new studies show.
Rat neurons thrive in a mouse brain world, testing ‘nature versus nurture’
Neurons from the two rodents can wire up together to form functional circuits—all while maintaining some species-specific properties, two new studies show.
It’s past time to stop using the Reading the Mind in the Eyes Test
The widely used measure of “theory of mind” needs to be re-examined, along with the long-standing claim that autism is linked to a lack of this ability.
It’s past time to stop using the Reading the Mind in the Eyes Test
The widely used measure of “theory of mind” needs to be re-examined, along with the long-standing claim that autism is linked to a lack of this ability.
Robots boost data consistency in rodent studies reliant on mechanical, optogenetic stimulation
Two new devices take experimenter variation out of the equation, the lead investigators say.
Robots boost data consistency in rodent studies reliant on mechanical, optogenetic stimulation
Two new devices take experimenter variation out of the equation, the lead investigators say.