cure-eb.org

Research summary infographic – click to see full-sized version

Mesenchymal Stromal Cells (Msc’s)

MSC’s are cells isolated from umbilical cord blood, bone marrow and other sources. Sometimes called stem cells, they can measure into different cell types and are able to stimulate anti inflammatory responses. Inflammation is considered a key factor in some of the complications that arise in Recessive Dystrophic Epidermolysis Bullosa (RDEB) such as poor wound healing, itching pain and importantly the particularly aggresive malignant skin cancer that develops.

 

Gene Editing

DEFINITION: Gene editing (or genome editing) is the insertion, deletion or replacement of DNA at a specific site in the genome of an organism or cell. It is usually achieved in the lab using engineered nucleases also known as molecular scissors. This work will not just benefit EB but will in theory be applicable to the people who suffer one of the estimated 5400 genetic disorders in the world, which accounts for 10% of people, 30 million in Europe alone.

BBC Health Report 1 Dec 2015 

 

Therapeutic Reprogramming Consortium

Read the full release HERE

Future projects

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In the Pipeline
    • Continuation of gene editing projects in the UK and US with a focus on getting to clinical trials both locally and whole body

 

    • Continuation of gene modified stem cell work aiming for clinical trials if preclinical research is supportive

 

    • Continuation of funding towards mesenchymal stromal cell production at Kings College London and treatments at Great Ormond Street Hospital

For Future Funding
    • Generation of gene modified skin grafts and clinical trial

 

    • RNA project

 

    • RDEB squamous cell carcinoma is a particularly aggressive malignant skin cancer. It has extremely high mortality and young adults with severe RDEB are at huge risk of developing it. We aim to pursue projects researching treatment options

 

    • Gene modification and gene editing approaches for Junctional EB

 

    • New project exploring novel treatment approaches for both local and systemic treatments including delivery mechanisms, skin substitutes and protein therapy

 

    • Dedicated GMP facilities for gene editing, gene modification and EB gene and cell therapy treatment suite

 

    • Lectureships in Dermatology, PhD studentships and Clinical Research fellowships