Story perspectives

Researchers Pursue Gene Therapy for Young Girl's Rare Disease

10/12/2025

25 5

Summary

Story summary

Researchers at the University of Queensland are developing a gene therapy for Tallulah Moon, a five-year-old with Hereditary Spastic Paraplegia Type 56 (SPG56).
Tallulah's parents founded the Genetic Cures for Kids foundation to pursue a cure.
Scientists have created brain organoids from Tallulah's skin cells to study the disease and test potential treatments.
They plan to deliver a functioning gene with a viral vector, but its effectiveness remains uncertain.

1 of 1

Story summary

Researchers at the University of Queensland are developing a gene therapy for Tallulah Moon, a five-year-old with Hereditary Spastic Paraplegia Type 56 (SPG56).
Tallulah's parents founded the Genetic Cures for Kids foundation to pursue a cure.
Scientists have created brain organoids from Tallulah's skin cells to study the disease and test potential treatments.
They plan to deliver a functioning gene with a viral vector, but its effectiveness remains uncertain.