John Robert Counsell

Refining gene delivery to skeletal muscle with a dual-strategy approach of muscle-tropic AAV capsids and muscle-specific promoters.

The paper investigates a dual-strategy approach using a muscle-tropic AAV capsid and muscle-specific promoter to improve gene delivery efficiency and reduce off-target expression in skeletal muscle.

Next Generation AAV-F Capsid gene therapy rescues disease pathology in a model of Pyruvate Dehydrogenase Complex Deficiency

This study compares AAV9 and AAV-F capsids for treating PDHD in mice, finding AAV-F improves survival and enzyme activity at lower doses but causes motor impairments.

Contaminating manufacturing plasmids and disrupted vector genomes present in liver tissue following adeno-associated virus gene therapy

The paper identifies manufacturing plasmid sequences and disrupted vector genomes in liver tissue from a patient with AAV gene therapy-related hepatitis.

GENE THERAPY PREVENTS HEPATIC MITOCHONDRIAL DYSFUNCTION IN MURINE DEOXYGUANOSINE KINASE DEFICIENCY

This study demonstrates that AAV9-mediated gene therapy prevents liver dysfunction in a mouse model of DGUOK deficiency.

Macrophage inhibitor clodronate enhances liver transduction of lentiviral but not AAV vectors or mRNA lipid nanoparticles in vivo.

This study shows that clodronate liposomes enhance lentiviral liver transduction but not AAV or LNP-mRNA in mice.

K978C CFTR restores essential epithelial function with greater efficiency than wildtype CFTR when expressed in CF airway cells.

This study shows that K978C CFTR, which increases open probability, restores epithelial function more efficiently than wildtype CFTR in CF airway cells when expressed in the presence of CF sputum.

Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors

The paper develops a method using adeno-associated virus vectors to generate somatic-transgenic mice for non-invasive monitoring of signaling pathways in disease models.