Custom-Made genetic drug targets rare blindness in single patient
NCT ID NCT07177196
First seen Nov 01, 2025 · Last updated May 12, 2026 · Updated 25 times
Summary
This study tests a custom-made genetic drug (antisense oligonucleotide) designed for one person with retinal dystrophy caused by a specific PRPH2 gene mutation. The goal is to see if the drug is safe and can slow or stop vision loss. Only one participant will receive the treatment, and the study focuses on safety and eye function changes.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for RETINAL DYSTROPHY are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
University of California San Diego
San Diego, California, 92093, United States
Conditions
Explore the condition pages connected to this study.