Epidermolysis Bullosa (EB) is a group of genetic adhesion disorders characterized by fragility of the skin. EB are devastating, highly disfiguring, blistering pathologies that severely impairs the quality of life of patients and affect approximately 500,000 people worldwide (www.debra.org).
Skin affected by Epidermolysis Bullosa
Patients with (non lethal) EB develop squamous cell carcinoma with high frequency and have a short life-span expectation. Epidermal stem cell-mediated gene therapy would be the only possible therapy, not only for genodermatoses, but also for other types of genetic skin diseases.
In 2006, our scientists published a proof of principle of the feasibility of ex-vivo gene therapy for laminin 332-dependent Junctional Epidermolysis Bullosa (JEB) by means of transplantation of autologous epidermal sheets prepared from genetically corrected epidemal stem cells. This clinical trial showed that ex vivo gene therapy of JEB is feasible and leads to full functional correction of the disease.
A cultured sheet of genetically corrected epidermis
Holostem Terapie Avanzate is currently running two Phase I/II clinical trials for combined ex-vivo cell and gene therapy for laminin 332-Junctional and Recessive Dystrophic EB, implementing a Phase I/II clinical trial for Col17-JEB and developing new gene-editing technologies for the treatment of dominant forms of EB, in collaboration with the Centre for Regenerative Medicine "Stefano Ferrari".
FOR EPIDERMOLYSIS BULLOSA
Provide patients suffering from
severe or rare, untreatable diseases
with stem cell-based advanced therapies
through ethical and scientific excellence
Tissue integrity and regeneration depend on a population of self-renewing somatic stem cells, above all in constantly renewing tissues. Regenerative Medicine refers to innovative therapies aimed at the permanent restoration of diseased tissues and organs.
Autologous cultures of limbal stem cells are routinely used for corneal regeneration and restoration of visual acuity in patients with severe corneal chemical and thermal burns associated to total unilateral or severe bilateral limbal stem cell deficiency.
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