FSU | Florida Institute for Pediatric Rare Diseases

FLORIDA STATE UNIVERSITY

The official logo for the Florida Institute for Pediatric Rare Diseases at the FSU College of Medicine. The design features the classic "FSU" block lettering separated by a vertical line from the institute's name in garnet text.
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2026 Research

RARE DISEASE RESEARCH & TRAINING

A group of researchers in white lab coats posing in a laboratory

Development of CRISPR/Cas Adenosine Base Editors for Reversing a Tumor Suppressor Gene Mutation in a Rare Pediatric Brain Tumor

ATRT Overview

ATRT Pediatric Brain Cancer

Three researchers in a laboratory, including one in a yellow isolation gown using a pipette to transfer liquid into a small tube while two colleagues observe closely.

A Novel Intranasal Protein-Based Therapeutic for Fragile X Syndrome

Fragile X Syndrome Overview

Fragile X syndrome

Jarrod Mousa, Ph.D., smiling in a laboratory in front of shelves filled with medical plates.

Structural Mapping of Human Antibody Responses to AAV in Pediatric Rare Disease Gene Therapy

Gene Therapy Overview

Inflammatory Reponse to Gene Therapy

A group photo of five Brian Miller lab members standing in two rows against a grey wall.

Hexokinase Domain Containing Protein 1 (HKDC1) Dysfunction in Pediatric Retinitis Pigmentosa

Pigmentosa Overview

HKDC1 Retinitis Pigmentosa

A man with dark hair, glasses, and a beard wearing a white button-down shirt sits at a laboratory workbench equipped with a computer monitor, scientific instruments, and various wires and cables.

Emerging Sarcomere Modulator for the Treatment of Pediatric Dilated Cardiomyopathy

Cardiomyopathy Overview

Pediatric Cardiomyopathy