Table 2.
Approach | Strategies | Current Status+ |
---|---|---|
Cell-based therapies | • Injection of allogeneic fibroblasts • Systemic or perilesional administration of mesenchymal stem cells • Autologous application of revertant mosaic cells • Use of cord blood stem cells |
CT CT CT PC |
Bone marrow transplantation | • BMT following complete myeloablation • Non-myeloablative conditioning • Autologous induced pluripotent stem cells |
CT CT PC |
Gene therapy/mRNA editing | • Ex vivo keratinocyte therapy • CRISPR/cas editing • RNA trans-splicing • PTC read-through and NMD antagonists |
CT PC PC PC |
Protein replacement therapy | • Delivery of recombinant type VII collagen in RDEB | PC |
Novel and repurposed drug treatments | • Anti-itch medications • Anti-fibrotic molecules (Losartan and Ruxolitinib) • Anti-inflammatory therapies • Enhanced wound healing (cathelicidin, Zorblisa, Keragel™) |
PS PC PS PS |
CT, clinical trials initiated, ongoing or recently completed; PC, these approaches are tested in preclinical studies, often utilizing appropriate mouse models of EB; PS, testing of these drugs is at the planning stages; BMT, bone marrow transplantation; PTC, premature termination codon; NMD, nonsense-mediated mRNA decay.
For details on ongoing clinical trials; see Supplemental Figure S1.