Section 58
Chapter 57,370

Cell therapy for basement membrane-linked diseases

Nyström, A.; Bornert, O.; Kühl, T.

Matrix Biology Journal of the International Society for Matrix Biology 57-58: 124-139


ISSN/ISBN: 1569-1802
PMID: 27609402
DOI: 10.1016/j.matbio.2016.07.012
Accession: 057369492

Download citation:  

For most disorders caused by mutations in genes encoding basement membrane (BM) proteins, there are at present only limited treatment options available. Genetic BM-linked disorders can be viewed as especially suited for treatment with cell-based therapy approaches because the proteins that need to be restored are located in the extracellular space. In consequence, complete and permanent engraftment of cells does not necessarily have to occur to achieve substantial causal therapeutic effects. For these disorders cells can be used as transient vehicles for protein replacement. In addition, it is becoming evident that BM-linked genetic disorders are modified by secondary diseases mechanisms. Cell-based therapies have also the ability to target such disease modifying mechanisms. Thus, cell therapies can simultaneously provide causal treatment and symptomatic relief, and accordingly hold great potential for treatment of BM-linked disorders. However, this potential has for most applications and diseases so far not been realized. Here, we will present the state of cell therapies for BM-linked diseases. We will discuss use of both pluripotent and differentiated cells, the limitation of the approaches, their challenges, and the way forward to potential wider implementation of cell therapies in the clinics.

PDF emailed within 0-6 h: $19.90