Gene therapy

Recent articles

Research image of SCN2A neurons.

Boosting SCN2A expression reduces seizures in mice

A modified form of CRISPR amps up expression of the gene—a strategy that could apply to other gene variations linked to autism.

By Charles Q. Choi
9 October 2025 | 5 min read
A clinician holds a clipboard while someone else sits on a couch.

Expediting clinical trials for profound autism: Q&A with Matthew State

Aligning Research to Impact Autism, a new initiative funded by the Sergey Brin Family Foundation, wants to bring basic science discoveries to the clinic faster.

By Lauren Schenkman
24 April 2025 | 8 min read
Research image of two mouse brain slices.

Split gene therapy delivers promise in mice modeling Dravet syndrome

The new approach overcomes viral packaging limitations by delivering SCN1A piecemeal and stitching it together in target cells.

By Holly Barker
10 April 2025 | 5 min read
A mouse sits on a gloved hand.

Molecular changes after MECP2 loss may drive Rett syndrome traits

Knocking out the gene in adult mice triggered up- and down-regulated expression of myriad genes weeks before there were changes in neuronal function.

By Chloe Williams
20 March 2025 | 5 min read
Photograph of a syringe.

What’s next for brain-directed gene therapy after death in Neurogene trial

The incident highlights that viral vectors can trigger deadly immune responses even when delivered directly to the nervous system.

By Calli McMurray
26 November 2024 | 6 min read

Expanding set of viral tools targets almost any brain cell type

Harmless viruses that encase short noncoding DNA elements called enhancers enable cell-type-specific gene delivery across the central nervous system in rodents and primates.

By Holly Barker
19 November 2024 | 2 min watch
A hand holds a stack of speech bubbles.

Leveraging the power of community to strengthen clinical trials for rare genetic syndromes

Families can become not only participants but champions of these research efforts.

By Shafali Spurling Jeste
11 July 2024 | 7 min read
Research image of two fluorescent-stained organoids.

CRISPR gives autism-linked genes a boost, rescues functioning

A modified form of the gene-editing tool increases the expression of CHD8 and SCN2A, showing potential for autism therapies.

By Giorgia Guglielmi
23 May 2024 | 5 min read
A digital rendering of a strand of RNA

New RNA editor boasts increased versatility, safety

The “PRECISE” technique reprograms cells in a way that, unlike DNA editors, avoids potentially permanent off-target effects.

By Charles Q. Choi
11 April 2024 | 4 min read
A slice of a cerebellum.

Mutation in top autism-linked gene may alter eye reflex

The discovery could help clinicians diagnose children who carry mutations in the gene, called SCN2A, and gauge their responses to potential therapies.

By Charles Q. Choi
26 February 2024 | 5 min read

Explore more from The Transmitter

Nachum Ulanovsky sits against a black background with one bat in his hands and another with its wings spread above his head.

Diving in with Nachum Ulanovsky

With an eye toward realism, the neuroscientist, who has a new study about bats out today, creates microcosms of the natural world to understand animal behavior.

By Claudia López Lloreda
16 October 2025 | 14 min listen
Developing human fetus.

Gene-activity map of developing brain reveals new clues about autism’s sex bias

Boys and girls may be vulnerable to different genetic changes, which could help explain why the condition is more common in boys despite linked variants appearing more often in girls.

By Giorgia Guglielmi
16 October 2025 | 6 min listen
Research image of astrocytic activation in mice.

Engrams in amygdala lean on astrocytes to solidify memories

Disrupting the astrocyte-neuronal dynamic in mice destabilizes their memory of fear conditioning.

By Lauren Schneider
15 October 2025 | 6 min listen

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