Aptamers in Basic Research, Neuroregeneration and Cancer
DNA aptamers for treatment of neurological diseases
Dr. Maher became involved in multiple sclerosis (MS) research through an invitation from Mayo Clinic neurologists. They asked him to conceive and supervise an in vitro selection approach to identify single-stranded DNA aptamers specific for myelin, seeking to mimic properties of autoantibodies previously investigated at Mayo Clinic. Studies with lead molecules in this series show remyelination activity in the TMEV mouse model of MS when animals were treated with one of the aptamers as a tetrameric conjugate. Subsequent collaborations have led to funded work to seek DNA aptamers that promote neuron health in models of amyotrophic lateral sclerosis.
DNA aptamers targeting sub-cellular compartments
The Nucleic Acid Structure and Recognition Lab is pioneering novel approaches to identify unusual DNA oligonucleotides that penetrate membranes of living cells and reach subcellular compartments. Such oligonucleotides could provide new approaches to delivering drugs and macromolecules to living cells. Example applications include funded collaborative work using in vivo aptamer selections to direct toxins to glioblastoma tumors.