In pursuit of gene therapies for autism-linked conditions

Recent articles

Research image of gene-edited mouse liver cells compared to controls.

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.

By Charles Q. Choi
27 June 2023 | 5 min read
Long-tailed macaque in the canopy of a fruiting strangler fig tree, surrounded by ripening figs. Gunung Palung National Park, Borneo.

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.

By Brendan Borrell
7 April 2023 | 5 min read
Illustration of a single patient in a clinical trial sitting on a bed in a glitchy, imperfectly rendered room.

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.

By Peter Hess
30 March 2023 | 7 min read
Jeremy Levin, chief executive officer of Ovid Therapeutics, stands against a bench in a lab.

What next for Angelman?

A meeting in Texas reckons with the future of treatment, following two setbacks in 2020.

By Angie Voyles Askham
20 October 2022 | 17 min listen
An Ultragenyx employee removes materials from a lab freezer.

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.

By Angie Voyles Askham
5 August 2022 | 4 min read

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.

By Holly Barker
14 January 2022 | 3 min read
lab technician pipetting in dramatic light

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.

By Angie Voyles Askham
16 June 2021 | 7 min read

New gene therapy methods deliver promise

Two unpublished studies detail improved techniques for delivering gene therapies to the brain.

By Angie Voyles Askham
14 January 2021 | 4 min read
Ralf Schmid, the research director in Neurodevelopmental diseases at the Gene Therapy Program Orphan Disease Center, loads replicated mouse dna into wells in the gel before applying electric current to test the viability of the dna.

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.

By Lydia Denworth, Brendan Borrell
14 October 2020 | 26 min listen

Explore more from The Transmitter

Research image of axons forming a path into brain metastases of small-cell lung cancer, integrating tumors into the brain.

Neurons fuel lung tumors that have spread to brain

Functional synapses between brain cells and cancer cells are key to the metastatic growth, according to new findings from two independent teams.

By Sarah DeWeerdt
6 October 2025 | 6 min read

Meet the Autism Data Science Initiative grantees

The awarded projects plan to study gene-and-environment interactions in people, stem cells and organoids, as well as predictors of positive life outcomes in autistic youth and adults.

By Calli McMurray
3 October 2025 | 2 min read
Stack of papers.

Exclusive: The 23 studies the FDA based its expanded leucovorin label on

The studies include 46 people, mostly toddlers, who have cerebral folate deficiency due to variants in a folate transporter.

By Claudia López Lloreda
2 October 2025 | 6 min read

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