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Identification of collagen type VII-producing cells in human bone marrow and cord blood

Completed
Project lead Dr Jakub Tolar
Organisation University of Minnesota
Project budget USD 221,380.00
Start date / Duration 01. Jun 2010 / 24 months
Funder(s) / Co-Funder(s) DEBRA Austria, DEBRA US, MSAP/EBEP Recommended
Research area Molecular therapy

Project details

Short lay summary

We have shown that transplatnation of bone marrow and cord blood helps patients with RDEB. The skin blisters and mouth sores healed and collagen type VII, which is deficient in RDEB, has appeared in skin after transplantation. We are excited that this has been possible, but we aim to provide even better treatment for people with RDEB. We reason that only some cells, perhaps a small minority infused with bone marrow and cord blood, do in fact helpt to heal the skin. Therefore, we thinkt that the more cells with capacity for skin and mucosal repair in RDEB we can provide, the better it will be for the patient. Thus, our primary aim is to identify the very cells in human bone marrow and cord blood responsible for the favorable outcome we have observed in our patients with RDEB after bone marrow and cord blod transplantation. As we are focused on clinical translation, we wish to study the role of human, rather than mouse, blood-forming cells in the healing of RDEB wounds. To that end we will use a unique cell transplantation mouse model, in which mice have human blood, thus mimicking human transplantation. To make this model relevant to RDEB, we will persist in mouse recipients unable to express C7. We will first identif the signals that attract bone marrow and cord blood cells to skin, and then we will assess whether these human cells can heal RDEB skin more rapidly and more completely than traditional bone marrow and cord blood transplantation. If successful, this method can be quickly brought to the clinic and have a significant positive impact on patients with RDEB.

Scientific summary

Our primary goal is to develop a treatment that produces whole-body healing for individuals with the inherited skin fragility disorder epidermolysis bullosa. We have shown that normal bone marrow transplantation provides type VII collagen-producing cells that migrate to the injured skin and mediate wound repair. With the funding from DebRA Austria, we were able to show the specific cells in the bone marrow that express type VII collagen. Furthermore, we know now how to isolate the groups of cells that are better at homing to injured skin than the rest of the blood-forming cells contained in the bone marrow and cord blood grafts. Our work to amplify these cells into clinically meaningful numbers is ongoing. In short, we have introduced an entirely novel concept of stem cell therapy whereby cells from one organ (bone marrow) are successfully employed for the tissue repair of another organ (skin). This paradigm opens never-before-imagined possibilities in regenerative medicine.


Keywords

Tolar
Bone marrow
cord blood
RDEB
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