DNA sequences

In-Silico DMD

  • Duchenne muscular dystrophy (DMD)
  • Gene therapies: 
    • Exon-skipping (PMOs)
    • Gene editing (gRNAs)
  • Challenges: Efficiency, Applicability, Delivery
  • In silico design and screening tool
  1. choose which target sites to test in vitro 
  2. design efficient oligonucleotides (PMOs)
  3. design cell penetrating peptides (CPPs) to deliver the PMOs into the cell nucleus (= PPMOs)
  4. expand CRISPR-Cas9 use into DMD with appropriate gRNAs

Transfer Learning

Transfer Learning (TL) approaches for splice site prediction:

  • Different representations (n-grams, Position Probability Matrix) 
  • Different algorithms (transfer K-Means)

Current Research:

  • A qualitative comparison of traditional and deep learning TL approaches

CRISPR-Cas9 gRNA efficiency prediction models