# Crossing the finish line towards a disease-modifying treatment for Angelman syndrome

## TL;DR

This paper discusses the potential for genetic therapies to treat Angelman syndrome and other neurodevelopmental disorders by examining ongoing clinical trials and identifying key insights for future treatments.

## Contribution

The paper provides a roadmap for developing disease-modifying therapies for single-gene neurodevelopmental disorders, focusing on Angelman syndrome.

## Key findings

- Ongoing clinical trials for Angelman syndrome may soon determine if restoring broken genes can mend derailed neurodevelopment.
- The paper identifies insights needed to accelerate the development of genetic therapies for neurodevelopmental disorders.
- Findings could serve as a model for treating other single-gene neurodevelopmental disorders.

## Abstract

Recent progress in the development of genetic therapies promises that impactful treatments for single-gene neurodevelopmental disorders are imminent. But can derailed neurodevelopmental processes be mended after broken genes are replaced or otherwise restored? The results of ongoing clinical trials for Angelman syndrome will soon yield answers to this pressing question, yet the trials face significant obstacles. Here we identify insights needed to aid the quest for a disease-modifying Angelman syndrome therapy, which could serve as a roadmap for the expeditious development of genetic therapies for other single-gene neurodevelopmental disorders.