JACK SULLIVAN, MD, PHD

Research Interest

Dr. Jack Sullivan, M.D., Ph.D. focuses his lab research on development of gene-based therapeutics for retinal and macular degenerations. In autosomal dominant genetic diseases post-transcriptional gene silencing (PTGS) agents, such as ribozymes or RNAi, can be used to suppress toxic target gene expression and ameliorate cellular stresses to promote increased functional vitality and survival. Such PTGS agents may also be used to alter normal gene expression systems (e.g., metabolic pathways) to slow the rate of temporally emergent diseases (e.g., age-related macular degeneration). Development of PTGS agents is befit with multi-level biocomplexity, which has markedly curtailed clinical translation of PTGS RNA drugs.

We developed an RNA Drug Discovery technology platform to relieve established bottlenecks that limit PTGS discovery. This platform allows rigorous evaluation of target mRNA accessibility, screening of many potential PTGS agents to identify a lead RNA drug candidate, optimization of this lead drug, and cellular toxicity determinations prior to animal experiments. We used this platform to develop potent ribozyme and RNAi agents against a particular disease target underlying autosomal dominant retinitis pigmentosa, and are now conducting a preclinical gene therapy trial of these agents in target-humanized mouse models of retinal degeneration. We are extending RNA Drug Discovery to validated targets in age-related macular degeneration.



Our studies employ a multidisciplinary approach of biophysics, bioengineering, molecular biology, molecular genetics, bioinformatics, and novel instrumentation and technology design. Our long-term goal is to translate successful RNA Drug Discovery into human clinical trials for people who suffer on a daily basis from visual loss due to these disease states.

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