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[Therapy for skin disease using bone marrow cells].

¹Department of Dermatology, Hokkaido University Graduate School of Medicine.

Nihon Rinsho Men'Eki Gakkai Kaishi = Japanese Journal of Clinical Immunology

|June 2, 2011

Summary

Bone marrow transplantation (BMT) shows promise for treating epidermolysis bullosa (EB) by using stem cell plasticity. This therapy led to improved skin structure and survival in mice with EB, suggesting potential for human treatment.

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Area of Science:

Regenerative Medicine
Dermatology
Stem Cell Biology

Background:

Congenital protein deficiencies, like epidermolysis bullosa (EB), present significant therapeutic challenges.
Previous attempts to treat such conditions leveraged stem cell plasticity, with limited success.
The role of bone marrow-derived cells in addressing keratinocyte structural protein defects remains an area of investigation.

Purpose of the Study:

To investigate the efficacy of bone marrow transplantation (BMT) as a treatment for epidermolysis bullosa (EB).
To determine if BMT can restore functional keratinocytes and improve skin integrity in a mouse model of EB.
To explore the potential of human cord blood cells in treating EB.

Main Methods:

Utilized collagen XVII (Col17) knockout mice as a model for epidermolysis bullosa.
Administered bone marrow transplantation (BMT) to adult Col17-deficient mice.
Analyzed donor-derived keratinocyte contribution, Col17 expression, hemidesmosomal structure, skin phenotype, and survival rates.
Transplanted human cord blood CD34+ cells into immunocompromised mice to assess differentiation potential.

Main Results:

BMT in Col17 knockout mice resulted in donor-derived keratinocytes and restored Col17 expression.
Significant improvements in hemidesmosomal structure and skin manifestations were observed post-BMT.
BMT treatment led to enhanced survival rates in the treated mice.
Human CD34+ cells successfully differentiated into keratinocytes expressing human skin proteins in vivo.

Conclusions:

Bone marrow transplantation (BMT) demonstrates significant potential as a systemic therapy for epidermolysis bullosa (EB).
The study validates the concept of using stem cell plasticity via BMT to address genetic defects in skin structural proteins.
Conventional BMT techniques offer a promising therapeutic avenue for human EB treatment.