Gene Therapy, Stem Cells: Prime for Vision Restoration

Editor's Note: The second installment of this five-part series, on hearing, will appear in the Oct. 1 issue. Using gene therapy, scientists earlier this year reversed blindness in three dogs afflicted with Leber congenital amaurosis (LCA). The news excited the scientific world and popular press. LCA is a rare, inherited disease characterized by a severe loss of vision at birth. Researchers at the University of Pennsylvania, Cornell University, and University of Florida showed that injecting a

Written byJennifer Fisher Wilson
| 7 min read

Register for free to listen to this article
Listen with Speechify
0:00
7:00
Share

This research characterizes the current state of scientific investigation into the world of blindness: virtually all human trials are far down the road, and any findings, so far, pertain only to the particular disease itself. A bright side does exist: vision restoration is a prime candidate for both gene therapy and stem cell transplants. And other researchers are using different approaches in which electrodes implanted on the retina allow blind people to see edges and shapes thanks to electrode stimulation from a camera mounted on a pair of glasses. 2 These researchers, from Johns Hopkins University, are awaiting Food and Drug Administration approval for a Phase I trial, says project director Mark Humayun. "Even restoring some vision in blind patients can have a significant impact on their lives by restoring their independent mobility," Humayun says.

The American Foundation for the Blind estimates that about 10 million people in the United ...

Interested in reading more?

Become a Member of

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

Meet the Author

Published In

Share
Illustration of a developing fetus surrounded by a clear fluid with a subtle yellow tinge, representing amniotic fluid.
January 2026, Issue 1

What Is the Amniotic Fluid Composed of?

The liquid world of fetal development provides a rich source of nutrition and protection tailored to meet the needs of the growing fetus.

View this Issue
Skip the Wait for Protein Stability Data with Aunty

Skip the Wait for Protein Stability Data with Aunty

Unchained Labs
Graphic of three DNA helices in various colors

An Automated DNA-to-Data Framework for Production-Scale Sequencing

illumina
Exploring Cellular Organization with Spatial Proteomics

Exploring Cellular Organization with Spatial Proteomics

Abstract illustration of spheres with multiple layers, representing endoderm, ectoderm, and mesoderm derived organoids

Organoid Origins and How to Grow Them

Thermo Fisher Logo

Products

Brandtech Logo

BRANDTECH Scientific Introduces the Transferpette® pro Micropipette: A New Twist on Comfort and Control

Biotium Logo

Biotium Launches GlycoLiner™ Cell Surface Glycoprotein Labeling Kits for Rapid and Selective Cell Surface Imaging

Colorful abstract spiral dot pattern on a black background

Thermo Scientific X and S Series General Purpose Centrifuges

Thermo Fisher Logo
Abstract background with red and blue laser lights

VANTAstar Flexible microplate reader with simplified workflows

BMG LABTECH