Jump to Main Content
- Schaffer, David, et al. Show all 3 Authors
- Current opinion in biotechnology 2019 v.60 pp. 99-103
- Dependoparvovirus; antibodies; capsid; engineering; gene therapy; immune response; lymphocytes; viruses
- ... Gene therapy is progressively emerging as a promising and powerful therapeutic modality, and adeno-associated virus (AAV) is a major delivery vehicle for such therapies. Among the most significant challenges that limit AAV’s utility, however, is the immune response it elicits. Antibodies elicited by prior exposure to natural virus or vector can bind to an AAV vector, preventing it from entering th ...
- Schaffer, David V., et al. Show all 2 Author
- Journal of Controlled Release 2016 v.240 pp. 287-301
- Dependovirus; clinical trials; gene therapy; gene transfer; models; neoplasm cells; neoplasms; transgenes
- ... Gene delivery vectors based on adeno-associated virus (AAV) have been utilized in a large number of gene therapy clinical trials, which have demonstrated their strong safety profile and increasingly their therapeutic efficacy for treating monogenic diseases. For cancer applications, AAV vectors have been harnessed for delivery of an extensive repertoire of transgenes to preclinical models and, mor ...
- PubMed Central:
- Schaffer, David V., et al. Show all 8 Authors
- Proceedings of the National Academy of Sciences of the United States of America 2009 v.106 no.10 pp. 3865-3870
- chlorides; cystic fibrosis; directed evolution; epithelium; extinction; gene therapy; gene transfer; humans; models; pathogenicity; pathogens; viruses
- ... Respiratory viruses evolve to maintain infectivity levels that permit spread yet prevent host and virus extinction, resulting in surprisingly low infection rates. Respiratory viruses harnessed as gene therapy vectors have illustrated this limitation. We used directed evolution in an organotypic human airway model to generate a highly infectious adeno-associated virus. This virus mediated gene tran ...
- PubMed Central: