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Gene & Cell Therapy in RDEB SCC
Patients with RDEB are at an increased risk of an aggressive squamous cell carcinoma (SCC) which is usually fatal. This project ...
Spray-on-Skin
Study of the Safety, Tolerability, and Efficacy of an iPS Cell-based Therapy for RDEB Delivered with a Spray-on-Skin Device ...
PASTE gene therapy in DEB
PASTE-Mediated Superexon Replacement of COL7A1 as a Treatment for Dystrophic Epidermolysis Bullosa ...
siRNA interference in EBS
siRNA-mediated allele-specific inhibition of mutant KRT5 and KRT14 transcripts carrying frequent dominant mutations in epidermolysis bullosa simplex (EBS) ...
Exon Skipping Approach to RDEB
Exon skipping approach to RDEB, with the aim to create a shorter but functional Collagen VII protein ...
Gabrielius
Gabrielius’ morning begins before 7AM, while he is still asleep. We start by changing the dressings on his legs. When ...
Rachel & Hon
I met Hon back in 2009. He was busy working for Whizz-Kids, teaching children how to use their wheelchairs. We ...
Kindler Syndrome
Kindler syndrome is a rare type of EB that results from mutations in the FERMT1 gene. From early infancy, people ...
EB Simplex
Epidermolysis Bullosa Simplex (EBS) Epidermolysis Bullosa Simplex is the most common type of EB, accounting for around 70% of cases ...
Junctional EB
Junctional Epidermolysis Bullosa (JEB) Junctional epidermolysis bullosa is recessively inherited and involves mutations in the genes for several components of ...
Dystrophic EB
Dystrophic EB (DEB) Dystrophic EB can be either dominantly or recessively inherited and involves defects in Type VII collagen. Blisters ...
Editing of RDEB fibroblasts
Testing the in vivo efficacy of base and prime editing in primary fibroblasts and in a 3D skin model of ...
Rigosertib for RDEB SCC
Rigosertib for recessive dystrophic epidermolysis bullosa-associated squamous cell carcinoma ...
Base editing of RDEB fibroblasts
Testing the efficacy of genomic base editing in dystrophic epidermolysis bullosa primary fibroblasts ...
Gene therapy for airway disease
Combined respiratory epithelial cell and gene therapy for amelioration of respiratory symptoms in children with Junctional Epidermolysis Bullosa ...
RDEB gene-edited skin
Pre-clinical and clinical trial for using gene-edited skin in Recessive Dystrophic Epidermolysis Bullosa ...
Mission EB
Double blind placebo controlled study of mesenchymal intravenous stromal cell infusions in children with Recessive Dystrophic Epidermolysis Bullosa ...
Next generation genome editing
Next generation genome editing for Recessive Dystrophic Epidermolysis Bullosa ...
Ocular stem cell regeneration
Stem cell regeneration of the ocular surface in Recessive Dystrophic Epidermolysis Bullosa ...
RDEB SCC gene sequencing
Deep sequencing Recessive Dystrophic Epidermolysis Bullosa squamous cell carcinoma ...
Lenticol F
Phase I study of lentiviral-mediated COL7A1 gene-modified autologous fibroblasts in adults with Recessive Dystrophic Epidermolysis Bullosa ...
Lenticol M
Mensenchymal stromal cells engineered to express Collagen VII for the treatment of Recessive Dystrophic Epidermolysis Bullosa ...
Limbal stem cells
Limbal stem cells for treatment of corneal wounds in Epidermolysis Bullosa Lay summary The cornea, the transparent outer surface of ...
TALEN-based gene editing
A TALEN-based approach to developing safer, more effective treatments for people with Epidermolysis Bullosa ...