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Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer.
We evaluated the safety and efficacy of an optimized adeno-associated virus (AAV; AAV2.RPE65) in animal models of the RPE65 form of Leber congenital amaurosis (LCA). Protein expression was optimizedExpand
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
Capsid decoys enhance the efficacy of AAV vector transduction after systemic delivery in the presence of neutralizing antibodies. A Slight of Hand for Gene Therapy Gene therapy has been quiteExpand
Viral vector manufacturing: quality attributes of rAAV used in clinical development.
TLDR
Recombinant Adeno-Associated Virus (rAAV) are widely used for human gene therapy, now the basis for two licensed products (for RPE65-/-, and SMA) and numerous other investigational treatments for a range of diseases including hemophilia, cystic fibrosis, muscular dystrophy & many neurological disorders. Expand
Optimizing Mobilization, Augmentation, and Gene Transfer of Multipotent Mesenchymal Stromal Cells (MSCs).
Aims: MSCs are cells being investigated for use in various therapies including facilitation of HSC transplantation and as gene therapy delivery vehicles. We have explored the potential to increaseExpand