Emerging gene therapeutics for epidermolysis bullosa under development

J Bischof, M Hierl, U Koller - International journal of molecular sciences, 2024 - mdpi.com
The monogenetic disease epidermolysis bullosa (EB) is characterised by the formation of
extended blisters and lesions on the patient's skin upon minimal mechanical stress. Causal …

Heterogeneity of reported outcomes in epidermolysis bullosa clinical research: a sco** review as a first step towards outcome harmonization

EWH Korte, T Welponer, J Kottner… - British Journal of …, 2023 - academic.oup.com
Background Epidermolysis bullosa (EB) is a rare, genetically and clinically heterogeneous
group of skin fragility disorders. No cure is currently available, but many novel and …

A scalable and cGMP-compatible autologous organotypic cell therapy for Dystrophic Epidermolysis Bullosa

G Neumayer, JL Torkelson, S Li, K McCarthy… - Nature …, 2024 - nature.com
Abstract We present Dystrophic Epidermolysis Bullosa Cell Therapy (DEBCT), a scalable
platform producing autologous organotypic iPS cell-derived induced skin composite (iSC) …

Paired nicking-mediated COL17A1 reframing for junctional epidermolysis bullosa

J Bischof, OP March, B Liemberger, SA Haas, S Hainzl… - Molecular Therapy, 2022 - cell.com
Junctional epidermolysis bullosa (JEB) is a debilitating hereditary skin disorder caused by
mutations in genes encoding laminin-332, type XVII collagen (C17), and integrin-α6β4 …

Highly efficient CRISPR/Cas9‐mediated exon skip** for recessive dystrophic epidermolysis bullosa

A du Rand, J Hunt, C Samson, E Loef… - Bioengineering & …, 2024 - Wiley Online Library
Gene therapy based on the CRISPR/Cas9 system has emerged as a promising strategy for
treating the monogenic fragile skin disorder recessive dystrophic epidermolysis bullosa …

Banana fruit (Musa sp.) DNA-magnetite nanoparticles: Synthesis, characterization, and biocompatibility assays on normal and cancerous cells

D Arregui-Almeida, M Coronel, K Analuisa… - Plos one, 2024 - journals.plos.org
Magnet-mediated gene therapy has gained considerable interest from researchers as a
novel alternative for treating genetic disorders, particularly through the use of …

Enhanced HDR-Mediated Correction of Heterozygous COL7A1 Mutations for Recessive Dystrophic Epidermolysis Bullosa

JMT Hunt, A du Rand, D Verdon, L Clemance… - … Therapy Nucleic Acids, 2025 - cell.com
Gene editing facilitated by homology-directed repair (HDR) holds great potential for treating
monogenetic disorders such as recessive dystrophic epidermolysis bullosa (RDEB) …

Current treatment landscape for dystrophic epidermolysis bullosa: from surgical management to emerging gene therapies and novel skin grafts

DF Villavisanis, DP Perrault, SN Kiani, D Cholok… - The Journal of Hand …, 2024 - Elsevier
Epidermolysis bullosa is a genetic skin disorder characterized by blister formation from
mechanical trauma. Dystrophic epidermolysis bullosa (DEB) is caused by mutations in the …

Epidermolysis bullosa

C Has, L Bruckner-Tuderman, J Uitto - Emery and Rimoin's Principles and …, 2025 - Elsevier
Epidermolysis bullosa (EB) is a heterogeneous group of genodermatoses characterized by
mucocutaneous blistering, resulting from intraepidermal or dermo-epidermal tissue …

A novel fluorescence-based screen of gene editing molecules for junctional epidermolysis bullosa

J Zwicklhuber, T Kocher, B Liemberger… - International Journal of …, 2023 - mdpi.com
Junctional epidermolysis bullosa (JEB) is a severe blistering skin disease caused by
mutations in genes encoding structural proteins essential for skin integrity. In this study, we …