RNA Writing Trans-Splicing for Enhancing Frataxin Translation
|Apr 7 2025
Characterization of ocular phenotype of the YG8-800 mouse model of FA and retinal-targeted treatment by AAV based CRISPR-Cas9 gene editing
|Jan 8 2025
Rescuing frataxin expression by alternative splicing reprogramming with base editing
|Dec 27 2024
Application of RSwitch technology to improve Friedreich’s ataxia viral gene therapy safety and efficacy: control of FXN transgene expression through a small molecule regulation
|Dec 5 2024
Development of Epigenetic Editors for Frataxin Reactivation
|Oct 30 2024
Enhancing Gene Therapy Delivery to the Brain via Human Blood-Brain Barrier Receptors
|Sep 27 2024
Shortening GAA repeats in Friedreich’s Ataxia
|Sep 24 2024
Postnatal development of the cerebellar circuitry in the KIKO mouse model of Friedreich’s ataxia
|Jul 31 2024
Deliverable in vitro transcribed mRNAs of Frataxin as a therapeutic approach for Friedreich ataxia
|Mar 23 2024
Paired Prime Editors to treat Friedreich’s Ataxia
|Mar 1 2024
Developing and delivering of novel gene editing technologies to contract GAA repeats in Friedreich’s ataxia
|Oct 19 2023
Hematopoietic Stem Cell Transplantation in FRDA: Proof-of-concept studies using novel conditioning regimen and the YG8s(GAA)>800 mouse