As a post-doctoral researcher and Fulford junior research fellow in the laboratory of Matthew Wood, I am involved in translational research for both Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA). Both disorders are treatable with splice switching oligonucleotides to correct genetic mutations. My focus is the targeted delivery of these therapies to skeletal muscle and central nervous system tissues, two tissues difficult to treat.
My PhD training was in the field of SMA at the Northwestern University. In this lab we developed several new lines of mice including an inducible line for early onset SMA to study disease pathology. Since coming to the Wood lab my work has focused on improving and designing new therapies for DMD and SMA. Over the past six years the Wood lab has used peptide delivery systems for improving the efficacy of antisense oligonucleotide therapies. I am currently translating this work into the SMA field.
Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment.
Betts CA. et al, (2019), Hum Mol Genet, 28, 396 - 406
Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment.
Gait MJ. et al, (2018), Nucleic Acid Ther
Interventions Targeting Glucocorticoid-Krüppel-like Factor 15-Branched-Chain Amino Acid Signaling Improve Disease Phenotypes in Spinal Muscular Atrophy Mice.
Walter LM. et al, (2018), EBioMedicine, 31, 226 - 242
Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy.
Shabanpoor F. et al, (2017), Nucleic Acid Ther, 27, 130 - 143
Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.
Hammond SM. et al, (2016), Proc Natl Acad Sci U S A, 113, 10962 - 10967