Rachel M. Bailey

A minimally modified AAV9 variant (AAV9fyhr) for enhanced CNS gene delivery by intra-CSF administration.

This paper describes a novel AAV9 variant, AAV9fyhr, that improves CNS gene delivery while minimizing peripheral tissue tropism through targeted amino acid modifications.

Preclinical Development of a Vectorized Artificial miRNA Gene Therapy for Tauopathies

This paper develops a gene therapy using artificial miRNA delivered via adeno-associated virus to reduce tau in a mouse model of tauopathy, showing efficacy even at late disease stages.

AAV-mediated gene therapy for SLC13A5 citrate transporter disorder rescues epileptic and metabolic phenotypes

This study shows that AAV-mediated gene therapy in SLC13A5-deficient mice reduces extracellular citrate, normalizes electrophysiologic and sleep issues, and improves seizure resistance.

Valine and Inflammation Drive Epilepsy in a Mouse Model of ECHS1 Deficiency

This paper develops a mouse model of ECHS1 deficiency and identifies valine metabolism and inflammation as drivers of epilepsy in the disease.

Nitrous Oxide Enhances Focused Ultrasound Mediated Delivery of Viral Gene Therapy

Nitrous Oxide reduces microbubble and ultrasound pressure requirements for BBB opening and improves AAV9 vector delivery.