Gene Therapy for Brain Disease

Delivering a missing enzyme to the brains of paralyzed children with a rare, life-threatening neurological disease restores movement and builds muscle mass.

| 2 min read

Register for free to listen to this article
Listen with Speechify
0:00
2:00
Share

SHUTTERSTOCK, IDESIGN

An inherited disorder known as aromatic L-amino acid decarboxylase deficiency, or AADC, leaves patients unable to produce the neurotransmitter dopamine, leaving them nearly paralyzed until they die in early childhood. But a new gene therapy, which involves the delivery of the missing L-amino acid decarboxylase enzyme that converts the chemical precursor L-DOPA to dopamine, helped four Taiwanese children, aged 4 to 6 years, move their heads and sit up on their own, according to a study published today (May 16) in Science Translational Medicine.

“The children in this study have the most severe form of inherited movement disorder known, and the only treatments so far have been supportive ones,” pediatric cardiologist Barry Byrne, director of the University of Florida’s Powell Gene Therapy Center, said in ...

Interested in reading more?

Become a Member of

The Scientist Logo
Receive full access to more than 35 years of archives, as well as TS Digest, digital editions of The Scientist, feature stories, and much more!
Already a member? Login Here

Keywords

Meet the Author

  • Jef Akst

    Jef Akst was managing editor of The Scientist, where she started as an intern in 2009 after receiving a master’s degree from Indiana University in April 2009 studying the mating behavior of seahorses.
Share
3D illustration of a gold lipid nanoparticle with pink nucleic acid inside of it. Purple and teal spikes stick out from the lipid bilayer representing polyethylene glycol.
February 2025, Issue 1

A Nanoparticle Delivery System for Gene Therapy

A reimagined lipid vehicle for nucleic acids could overcome the limitations of current vectors.

View this Issue
Enhancing Therapeutic Antibody Discovery with Cross-Platform Workflows

Enhancing Therapeutic Antibody Discovery with Cross-Platform Workflows

sartorius logo
Considerations for Cell-Based Assays in Immuno-Oncology Research

Considerations for Cell-Based Assays in Immuno-Oncology Research

Lonza
An illustration of animal and tree silhouettes.

From Water Bears to Grizzly Bears: Unusual Animal Models

Taconic Biosciences
Sex Differences in Neurological Research

Sex Differences in Neurological Research

bit.bio logo

Products

Photo of a researcher overseeing large scale production processes in a laboratory.

Scaling Lentiviral Vector Manufacturing for Optimal Productivity

Thermo Fisher Logo
Collage-style urban graphic of wastewater surveillance and treatment

Putting Pathogens to the Test with Wastewater Surveillance

An illustration of an mRNA molecule in front of a multicolored background.

Generating High-Quality mRNA for In Vivo Delivery with lipid nanoparticles

Thermo Fisher Logo
Tecan Logo

Tecan introduces Veya: bringing digital, scalable automation to labs worldwide