sample of publications

• articles 

  • Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors.  Molecular Therapy - Methods and Clinical Development.  27:96-108. 2022
  • Mechanistic interrogation of mutation-independent disease modulators of RDEB identifies the small leucine-rich proteoglycan PRELP as a TGF-beta antagonist and inhibitor of fibrosis.  MATRIX BIOLOGY.  111:189-206. 2022
  • Non-viral delivery of CRISPR-Cas9 complexes for targeted gene editing via a polymer delivery system.  GENE THERAPY.  29:157-170. 2022
  • Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing.  MOLECULAR THERAPY.  29:2008-2018. 2021
  • Efficient CRISPR-Cas9-mediated gene ablation in human keratinocytes to recapitulate genodermatoses: modeling of Netherton syndrome.  Molecular Therapy - Methods and Clinical Development.  18:280-290. 2020
  • Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing.  MOLECULAR THERAPY.  27:986-998. 2019
  • Deletion of a Pathogenic Mutation-Containing Exon of COL7A1 Allows Clonal Gene Editing Correction of RDEB Patient Epidermal Stem Cells.  Molecular Therapy Nucleic Acids.  11:68-78. 2018

• conference contributions 

  • CRISPR/Cas9-based gene editing strategies for clinically-relevant ex vivo correction of Recessive Dystrophic Epidermolysis Bullosa.  59-59. 2020
  • Development of a Netherton Syndrome model based on targeted deletion of SPINK5 in human keratinocytes.  S267-s267. 2019
  • Highly efficient gene-editing strategies for clinically-relevant ex vivo correction of Recessive Dystrophic Epidermolysis Bullosa in primary patient cells 2019
  • Preclinical models for in vivo gene editing of COL7A1 based on delivery of CRISPR/Cas9 to RDEB patient skin by adenoviral vectors 2019
  • Clinically-relevant correction of recessive dystrophic epidermolysis bullosa by dual sgRNA CRISPR/Cas9-mediated gene editing.  A116-A117. 2018
  • Gene editing-mediated excision of mutation-bearing exon 80 of COL7A1 gene for the efficient correction of recessive dystrophic epidermolysis bullosa patient derived-epidermalstem cells 2017

• theses 

  • GENE EDITING-BASED PROTOCOLS FOR THE EX VIVO CORRECTION OF RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA