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Review
. 2018 Oct;29(10):1098-1105.
doi: 10.1089/hum.2018.151.

Gene and Cell Therapy for Muscular Dystrophies: Are We Getting There?

Francesco Galli  1 , Laricia Bragg  1 , Linda Meggiolaro  1 , Maira Rossi  1 , Miriam Caffarini  1 , Naila Naz  1 , Sabrina Santoleri  1 , Giulio Cossu  1
Affiliations
Review

Gene and Cell Therapy for Muscular Dystrophies: Are We Getting There?

Francesco Galli et al. Hum Gene Ther. 2018 Oct.

Abstract

In the last few years, significant advances have occurred in the preclinical and clinical work toward gene and cell therapy for muscular dystrophy. At the time of this writing, several trials are ongoing and more are expected to start. It is thus a time of expectation, even though many hurdles remain and it is unclear whether they will be overcome with current strategies or if further improvements will be necessary.

Keywords: cell therapy; gene therapy; muscular dystrophy.

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Conflict of interest statement

No competing financial interests exist.

Figures

<b>Figure 1.</b>
Figure 1.
Similarity of delivery routes for adeno-associated vectors (left) or cells (right). In both cases, local transplantation (top) leads to a high concentration but only in the side of injection. In contrast vascular delivery (bottom) leads to widespread concentration, which however may not be sufficient for a significant therapeutic effect. The figure was produced using Servier Medical Art (www.servier.com).
See this image and copyright information in PMC

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