Publications

• A Compact Base Editor Rescues AATD-associated Liver and Lung Disease in Mouse Models

• A single amino acid variant in the variable region I of AAV capsid confers liver detargeting

• BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease

• Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy

• Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques

• Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor

• Serum Western Blot for the Detection of a c-Myc Protein Tag in Non-human Primates and Mice

• Approaches to Therapeutic Gene Editing in Alpha-1 Antitrypsin Deficiency

• Alpha-1 Antitrypsin Deficiency

• Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo

• Gene therapy for alpha-1 antitrypsin deficiency: an update

• Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts

• Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient

• Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells

• Therapeutic plasma exchange to mitigate flunixin meglumine overdose in a cria

• Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

• The rapidly evolving state of gene therapy

• Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study

• Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema

• Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency

• Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA

• Genotyping Protocol for the Alpha-1 Antitrypsin (PiZ) Mouse Model

• 5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency

• Retro-Orbital Venous Sinus Delivery of rAAV9 Mediates High-Level Transduction of Brain and Retina Compared with Temporal Vein Delivery in Neonatal Mouse Pups

• Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods

• Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency

• Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials

• Age dependence of lung mesenchymal stromal cell dynamics following pneumonectomy

• Gene transfer in skeletal and cardiac muscle using recombinant adeno-associated virus

• Gene transfer in the lung using recombinant adeno-associated virus

• Sustained miRNA-mediated knockdown of mutant AAT with simultaneous augmentation of wild-type AAT has minimal effect on global liver miRNA profiles

• Age-dependent decline in mouse lung regeneration with loss of lung fibroblast clonogenicity and increased myofibroblastic differentiation

• Urinary bladder agenesis in an alpaca (Vicugna pacos) cria

• A comparison of total, respirable, and real-time airborne particulate sampling in horse barns