Area of research
Molecular Biology · Genetics
Research interest
Research focused on Exon and Exon skipping, with related work in Duchenne muscular dystrophy, Neurofibromatosis, Dystrophin. Notable publications include 'Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases', 'Next-generation muscle-directed gene therapy by in silico vector design', and 'Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy'.
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model
Targeted exon skipping of NF1 exon 17 as a therapeutic for neurofibromatosis type I
Mutation-Directed Therapeutics for Neurofibromatosis Type I
Next-generation muscle-directed gene therapy by in silico vector design
A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures
Nuclear poly(A)-binding protein aggregates misplace a pre-mRNA outside of SC35 speckle causing its abnormal splicing
Genome-wide Computational Analysis Reveals Cardiomyocyte-specific Transcriptional Cis-regulatory Motifs That Enable Efficient Cardiac Gene Therapy
Long-Term Episomal Transgene Expression from Mitotically Stable Integration-Deficient Lentiviral Vectors
Chromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases