C. Liberati

Clinical Gene Therapy Trials for Pompe Disease

B. Byrne, M. Corti, C. Liberati, 2019, Muscle Gene Therapy.

Pulmonary embolism after intra-articular injection of methylprednisolone and hyaluronate.

G. Sebastiani, G. Famularo, S. Polchi, 2001, Clinical and experimental rheumatology.

Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease.

T. Conlon, B. Byrne, I. Tuna, 2017, Human gene therapy. Clinical development.

Androgen receptor polymorphism (CAG repeats) and androgenicity

A. Pinchera, E. Martino, D. Canale, 2005, Clinical endocrinology.

Preclinical Gene Therapy Studies for Metabolic Myopathy

B. Byrne, M. Corti, C. Liberati, 2019, Muscle Gene Therapy.

Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation

B. Byrne, I. Tuna, J. Lammers, 2023, Molecular therapy. Methods & clinical development.

Diaphragm pacing and independent breathing in individuals with severe Pompe disease

B. Byrne, Barbara K. Smith, D. Fuller, 2023, Frontiers in rehabilitation sciences.