Anthony P. DeFeo

Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I.

Synthetic zinc finger nuclease design and rapid assembly.

TALEN-based gene correction for epidermolysis bullosa.

Fanconi anemia gene editing by the CRISPR/Cas9 system.

Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases.

CRISPR/Cas9 Targeted Gene Editing and Cellular Engineering in Fanconi Anemia.

Angiotensin receptor blockade mediated amelioration of mucopolysaccharidosis type I cardiac and craniofacial pathology.

CRISPR/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosa.

CRISPR/Cas9-Mediated Correction of the FANCD1 Gene in Primary Patient Cells.

Correction of Fanconi Anemia Mutations Using Digital Genome Engineering.

Cas9-induced targeted integration of large DNA payloads in primary human T cells via homology-mediated end-joining DNA repair.

Targeting the intracellular immune checkpoint CISH with CRISPR-Cas9-edited T cells in patients with metastatic colorectal cancer: a first-in-human, single-centre, phase 1 trial.