Biology of Adeno-Associated Virus (AAV)
We are interested in the study of the biology of AAV transduction in all its aspects, including binding, entry, post-entry, replication, viral assembly and release, as well as AAV epidemiology and immunology.
We study the evolution of viruses currently in use for gene transfer applications. We hypothesize that the study of the natural history of the viruses on which vectors are based highlight underlying biology relevant to their application as vectors and fundamental findings in how viruses assemble, package DNA, and transduce a particular cell target.
Approaches to Deliver Large Therapeutic Expression Cassettes to the Retina
Current vector systems are limited in terms of how much genetic cargo they can effectively shuttle. We are interested in developing technologies to overcome this limitation.
Immunology of Retinal Gene Transfer
The eye is generally seen as an immune-privileged organ due to passive and active means of tolerance. Many gene therapy approaches rely on the delivery of a protein or vector system that is foreign to the host and may elicit an immune-response. Our laboratory studies and develops assays to monitor these processes in an effort to maximize the safety of the therapeutic strategy.
Genome Editing Therapy
While many ideas use genetic information to treat disease are built on adding, augmenting or silencing genes to the host cell, tissue or organ, we are also exploring ways that novel methods to somatically alter cellular genomic information could be used therapeutically in blindness.
Delivery of a gene to a cell is cornerstone of genetic therapy. Its efficiency, safety, specificity, longevity and many of the other parameters essential for a treatment are determined by the vector the gene is shuttled into the cell. Our laboratory continuously pursues the optimization of current vectors and the discovery and generation of new vector technologies.
Translational Gene Therapy Programs
Our laboratory is dedicated to use existing technologies into preclinical and clinical programs for indications with an unmet clinical need. As a key member of the Ocular Genomics Institute, we believe gene therapy in ophthalmology is:
- Leber Congenital Amaurosis
- Retinitis Pigmentosa
- Usher Syndrome