Research Portfolio

Liquid biopsy of EB blister fluid for stem cell isolation and development of gene therapy strategies

Development of an eye treatment for ocular surface damage

A medical grade nanoneedle bandage for topical COL7A1 gene therapy

Chemoprevention of recessive dystrophic epidermolysis bullosa-associated squamous cell carcinoma

Replay next-generation gene therapy for DEB

An opthalmic solution for the treatment of corneal abrasions associated with epidermolysis bullosa

Evaluation of long-term effect on skin homeostasis and regeneration in a RDEB mouse model using different delivery methods of LNPs encapsulating mRNA-based base editing approach

Bespoke antisense oligomer-mediated splice modulating therapies for Recessive Dystrophic Epidermolysis Bullosa patients

Towards cell and gene therapy for Junctional Epidermolysis Bullosa airway disease

Development of NvA-011 for topical application in Dystrophic Epidermolysis Bullosa

Base and prime editing strategies for Recessive Dystrophic Epidermolysis Bullosa

Gene & Cell Therapy in Recessive Dystrophic Epidermolysis Squamous Cell Carcinoma

Nonsense Tx reading through disease causing nonsense codons

PASTE-Mediated Superexon Replacement of COL7A1 as a Treatment for Dystrophic Epidermolysis Bullosa

Study of the Safety, Tolerability, and Efficacy of an iPS Cell-based Therapy for Recessive Dystrophic Epidermolysis Bullosa Delivered with a Spray-on-Skin Device

siRNA-mediated allele-specific inhibition of mutant KRT5 and KRT14 transcripts carrying frequent dominant mutations in Epidermolysis Bullosa Simplex

Testing the in vivo efficacy of base and prime editing in primary fibroblasts and in a 3D skin model of dystrophic epidermolysis bullosa

Rigosertib for recessive dystrophic epidermolysis bullosa-associated squamous cell carcinoma

Testing the efficacy of genomic base editing in dystrophic epidermolysis bullosa primary fibroblasts

Development of a topical gene-editing treatment to restore Collagen VII

Combined respiratory epithelial cell and gene therapy for amelioration of respiratory symptoms in children with Junctional Epidermolysis Bullosa

Pre-clinical and clinical trial for using gene-edited skin in Recessive Dystrophic Epidermolysis Bullosa

A nanoneedle bandage for topical intracellular COL7A1 gene therapy to accelerate wound healing in Recessive Dystrophic Epidermolysis Bullosa

Exon skipping approach to Recessive Dystrophic Epidermolysis Bullosa

Development of a topical gel formulation of decorin to reduce fibrosis

Double blind placebo controlled study of mesenchymal intravenous stromal cell infusions in children with Recessive Dystrophic Epidermolysis Bullosa

Development of IV-administered siRNAs targeting mutant KRT5 and KRT1 in EB Simplex

Development of an oral formulation of the peptide angiotensin-(1-7) to reduce fibrosis

Phase I/II ex vivo gene therapy clinical trial for RDEB using autologous skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector

Gene editing for Epidermolysis Bullosa

Next generation genome editing for Recessive Dystrophic Epidermolysis Bullosa

Epidermolysis Bullosa Therapeutic Reprogramming Consortium

Stem cell regeneration of the ocular surface in Recessive Dystrophic Epidermolysis Bullosa

Deep sequencing Recessive Dystrophic Epidermolysis Bullosa squamous cell carcinoma

Mensenchymal stromal cells engineered to express Collagen VII for the treatment of Recessive Dystrophic Epidermolysis Bullosa

Phase I study of lentiviral-mediated COL7A1 gene-modified autologous fibroblasts in adults with Recessive Dystrophic Epidermolysis Bullosa

A phase I/II study to evaluate allogeneic mesenchymal stromal cells for the treatment of skin disease in adults with Recessive Dystrophic Epidermolysis Bullosa

Limbal stem cells for treatment of corneal wounds in Epidermolysis Bullosa

A TALEN-based approach to developing safer, more effective treatments for people with Epidermolysis Bullosa

Clinical trial of mesenchymal stromal cells in Recessive Dystrophic Epidermolysis Bullosa