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Lab image titled "Gene & Cell therapy for RDEB SCC"

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

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 gene therapy in DEB

PASTE-Mediated Superexon Replacement of COL7A1 as a Treatment for Dystrophic Epidermolysis Bullosa ...
siRNA interference in EBS

siRNA interference in EBS

siRNA-mediated allele-specific inhibition of mutant KRT5 and KRT14 transcripts carrying frequent dominant mutations in epidermolysis bullosa simplex (EBS) ...
Dr Matthias Titeux & Prof Alain Hovnanian standing in a lab

Exon Skipping Approach to RDEB

Exon skipping approach to RDEB, with the aim to create a shorter but functional Collagen VII protein ...
Sohana

Sohana

My Day by Sohana At 8am I wake up to take five different medications and allow them to set in ...
Poppy

Poppy

Mornings are getting tougher the older she gets. Sleep is constantly broken throughout the night when she is uncomfortable or ...
Albi

Albi

Our little boy Albi was born with severe Recessive Dystrophic Epidermolysis Bullosa ...
Gabrielius

Gabrielius

Gabrielius’ morning begins before 7AM, while he is still asleep. We start by changing the dressings on his legs. When ...
Mason

Mason

Mason gets up for school about 7am when his respite nurse leaves. We will do any dressings that need doing ...
RAchel and Hon

Rachel & Hon

I met Hon back in 2009. He was busy working for Whizz-Kids, teaching children how to use their wheelchairs. We ...
Dylan

Dylan

By Karen, Dylan's mum After a healthy and low risk pregnancy, our beautiful son Dylan was born in June 2017 ...
Photo of Jenna & her family

Jenna

I grew up in a family where I was the first to be diagnosed with EB Simplex (EBS). I never ...
Kindler Syndrome

Kindler Syndrome

Kindler syndrome is a rare type of EB that results from mutations in the FERMT1 gene. From early infancy, people ...
EB Simplex

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 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

Dystrophic EB (DEB) Dystrophic EB can be either dominantly or recessively inherited and involves defects in Type VII collagen. Blisters ...
Isla

Isla

Isla's Day We turn off Isla’s feeding pump and give her her morning meds at about 7:30am and then wake her as ...
Editing of RDEB fibroblasts

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 RDEB SCC

Rigosertib for recessive dystrophic epidermolysis bullosa-associated squamous cell carcinoma ...
Jackson

Jackson

My son was not diagnosed with EB at birth. We noticed his first blisters at 4 months old. I had ...
Photo of Jennifer wearing a hat and sunglasses, with hills in the background

Jennifer

When I was born in 1972, not much about EB was known. No one else in my family had it ...
Ala'a

Ala’a

Living with EB has taught me how to be strong and tolerate severe pain. Therefore, it will not stop me ...
Vie

Vie

Finally, just before I turned 28, I was diagnosed with EB. And my world opened up! I was finally part ...
Allison

Allison

I gave the faulty gene to my lovely son. He is in constant pain but smiles through it - he ...
Female scientist working in lab

Base editing of RDEB fibroblasts

Testing the efficacy of genomic base editing in dystrophic epidermolysis bullosa primary fibroblasts ...
BrB-101

BrB-101

Development of a topical gene-editing treatment to restore Collagen VII ...
Gene therapy for airway disease

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

RDEB gene-edited skin

Pre-clinical and clinical trial for using gene-edited skin in Recessive Dystrophic Epidermolysis Bullosa ...
NanoCol

NanoCol

A nanoneedle bandage for topical intracellular COL7A1 gene therapy to accelerate wound healing in Recessive Dystrophic Epidermolysis Bullosa ...
Mission EB

Mission EB

Double blind placebo controlled study of mesenchymal intravenous stromal cell infusions in children with Recessive Dystrophic Epidermolysis Bullosa ...
FIBRX Derm

FIBRX Derm

Development of a topical gel formulation of decorin to reduce fibrosis ...
RNA Derm

RNA Derm

Development of IV-administered siRNAs targeting mutant KRT5 and KRT1 in EB Simplex ...
TXA127

TXA127

Development of an oral formulation of the peptide angiotensin-(1-7) to reduce fibrosis ...
EBGRAFT

EBGRAFT

Phase I/II ex vivo gene therapy clinical trial for RDEB using autologous skin equivalent grafts genetically corrected with a COL7A1-encoding ...
Gene editing for EB

Gene editing for EB

Gene editing for Epidermolysis Bullosa ...
EB iPS Consortium

EB iPS Consortium

Epidermolysis Bullosa Therapeutic Reprogramming Consortium ...
Next generation genome editing

Next generation genome editing

Next generation genome editing for Recessive Dystrophic Epidermolysis Bullosa ...
Ocular stem cell regeneration

Ocular stem cell regeneration

Stem cell regeneration of the ocular surface in Recessive Dystrophic Epidermolysis Bullosa ...
RDEB SCC gene sequencing

RDEB SCC gene sequencing

Deep sequencing Recessive Dystrophic Epidermolysis Bullosa squamous cell carcinoma ...
Lenticol F

Lenticol F

Phase I study of lentiviral-mediated COL7A1 gene-modified autologous fibroblasts in adults with Recessive Dystrophic Epidermolysis Bullosa ...
Lenticol M

Lenticol M

Mensenchymal stromal cells engineered to express Collagen VII for the treatment of Recessive Dystrophic Epidermolysis Bullosa ...
ADSTEM

ADSTEM

A phase I/II study to evaluate allogeneic mesenchymal stromal cells for the treatment of skin disease in adults with Recessive ...
Limbal stem cells

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

TALEN-based gene editing

A TALEN-based approach to developing safer, more effective treatments for people with Epidermolysis Bullosa ...
EBSTEM

EBSTEM

Clinical trial of mesenchymal stromal cells in Recessive Dystrophic Epidermolysis Bullosa ...
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  • Completed Projects
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  • Faces of EB
  • Gene Editing
  • Gene Therapy
  • Quality of Life Change
  • Symptom Relief
  • Types of EB
  • Whole Body Treatment
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Cell Therapy, Completed Projects, Quality of Life Change
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Cell Therapy, Completed Projects, Quality of Life Change
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Current Projects, Gene Therapy, Quality of Life Change
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Current Projects, Drug Therapy, Symptom Relief
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Current Projects, Drug Therapy, Whole Body Treatment
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Current Projects, Gene Therapy, Quality of Life Change
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Current Projects, Gene Therapy, Quality of Life Change, Whole Body Treatment