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Guanidyl-rich highly branched poly(β-amino ester)s for the delivery of dual CRISPR ribonucleoprotein for efficient large DNA fragment deletion.
J Control Release
January 2025
Charles Institute of Dermatology, School of Medicine, University College Dublin, Dublin, Ireland. Electronic address:
Gene editing technologies, particularly clustered regularly interspersed short palindromic repeats (CRISPR) and CRISPR-associated (Cas) proteins, have revolutionized the ability to modify gene sequences in living cells for therapeutic purposes. Delivery of CRISPR/Cas ribonucleoprotein (RNP) is preferred over its DNA and RNA formats in terms of gene editing effectiveness and low risk of off-target events. However, the intracellular delivery of RNP poses significant challenges and necessitates the development of non-viral vectors.
View Article and Find Full Text PDFLived experiences of patients with epidermolysis bullosa: A rare genetic skin disease.
Health SA
December 2024
Department of Clinical Psychology, Faculty of Health Sciences, Greys Hospital, Pietermaritzburg, South Africa.
Background: Epidermolysis bullosa (EB) is a rare genodermatosis that results in extreme skin fragility, for which there is no cure and may be fatal. The quality of life of patients affected may be greatly impacted.
Aim: This study aims to understand the lived experiences of patients with EB.
Long-term safety and efficacy of Oleogel-S10 (birch bark extract) in epidermolysis bullosa: 24-month results from the Phase III EASE Study.
Br J Dermatol
January 2025
The School of Translational Medicine, Monash University and Alfred Health, Melbourne VIC, Australia.
Background: Epidermolysis bullosa (EB) is a group of rare, severe, genetic disorders characterised by persistent skin fragility and open wounds. EB manifests as cutaneous and mucosal blistering, erosions and impaired wound healing.
Objectives: To determine the long-term efficacy, tolerability and safety of Oleogel-S10 (birch bark extract) in dystrophic (DEB) and junctional (JEB) EB in the 24-months open-label phase (OLP) of the EASE study.
Colchicine as a treatment option for inherited epidermolysis bullosa.
Clin Exp Dermatol
January 2025
Department of Dermatology, St George Hospital, Sydney, NSW, Australia.
Weekly Intraperitoneal Injection of Tamoxifen in an Inducible In Vivo Model of Junctional Epidermolysis Bullosa Generates Early and Advanced Disease Phenotypes.
JID Innov
March 2025
Cell Biology & Cutaneous Research, Blizard Institute, Queen Mary University of London, London, United Kingdom.
Junctional epidermolysis bullosa caused by loss-of-function variants in genes encoding the skin basement membrane proteins laminin 332, type XVII collagen, or integrin α6β4 affects patients from birth with severe blistering, eventually leading to scarring and early lethality. In this study, we have optimized a previously published junctional epidermolysis bullosa-knockout mouse model with weekly tamoxifen intraperitoneal injections, resulting in a more controllable and severe model. Owing to the titratable dosing, this model now recapitulates both early and advanced stages of the human disease, strengthening its use in therapeutic studies.
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