Table 1. Overview of the studies on Duchenne’s muscular dystrophy included in the systematic review.
| Authors | Year | Disease | Origin of model | Delivery to cells | Key-point(s): |
|---|---|---|---|---|---|
| Amoasi et al. [39] | 2017 | DMD*† | ΔEx50 M | AAV 9 | New mouse model: ΔEx50. Early delivery increased editing-efficiency of exon 51 |
| Bengtsson et al. [59] | 2017 | DMD*† | mdx4cv M | EP, AAV 6 | Excised exon 52&53. Dual-, compared to single-, vector delivery was more efficient |
| Duchene et al. [60] | 2018 | DMD* | H | LF 2000 | Protocol for DMD correction, using the CinDel Method |
| Ehrke-Schulz et al. [57] | 2017 | DMD* | H | HC-AdV | Designed a new viral system (HC-Adv), with a large delivering capacity (35 kb) |
| El Refaey et al. [49] | 2017 | DMD*† | mdx/Utr+/− M | EP, AAV rh74 | Demonstrated functional improvement in cardiac contractility (papillary muscle) |
| Iyombe-Engembe et al. [61] | 2016 | DMD*† | H, hDMD/mdx M | LF 2000, EP | Deleted exons 51–53, creating a hybrid exon 50–54 in mice that contain human DMD |
| Kyrychenko et al. [46] | 2017 | DMD* | H | NF | Editing the ABD-1 domain showed improvement in functionality of cardiomyocytes |
| Lattanzi et al. [45] | 2017 | DMD* | H | LV | Demonstrated a strategy for editing the exon 2 duplication using one gRNA |
| Lee et al. [64] | 2017 | DMD*† | mdx M | CGNP | Induced HDR, delivering the CRISPR components with new Gold-nanoparticles. |
| Li et al. [56] | 2015 | DMD* | H | EP | Exon 44 skipping, frameshifting, and knockin. Knockin restored full protein length |
| Liao et al. [66] | 2017 | DMD† | Cas9/mdx, mdx M | AAV 9 | Epigenetically up-regulated expression of utrophin, using dead cas9 (dCas9-VP160) |
| Long et al. [65] | 2014 | DMD† | mdx M | MI | Germline editing in mice produced mosaic animals with 2–100% DMD correction |
| Long et al. [50] | 2016 | DMD† | mdx M | AAV 9 | Different modes of AAV 9 delivery show varying efficiency in restoring dystrophin |
| Maggio et al. [62] | 2016 | DMD* | H | AdV | Excised a large region encompassing exons 44–54, covering many mutations |
| Maggio et al. [58] | 2016 | DMD* | H | AdV | Explored editing strategies, using CRISPR-cas alone and combined with TALENs |
| Mou et al. [52] | 2017 | DMD* | M | LV | Used single gRNA skipping of exon 23 in C2C12 mouse cells. |
| Nelson et al. [53] | 2016 | DMD† | mdx M | AAV 8 | Deletion of exon 23 improved muscle function in both adult and neonatal mice |
| Ousterout et al. [44] | 2015 | DMD* ‡ | H in M | LF & EP | Edited patient myoblasts, engrafted in immunodeficient mice, restored dystrophin |
| Perrin et al. [67] | 2017 | DMD*† | Rag/mdx M | TransfeX, LF & EP | Increased the expression of Laminin subunit α1, using dCas9-VP160 |
| Tabebordbar et al. [54] | 2016 | DMD*† | mdx M | AAV 9 | Gene modifications possible in terminally differentiated muscle cells |
| Wojtal et al. [47] | 2016 | DMD* | H | EP | Increased the expression of utrophin, using dCas9-VP160 |
| Xu et al. [48] | 2016 | DMD*† | mdx M | EP, AdV | Deletion of exons 21–23 improved sarcolemal integrity in skeletal muscle cells |
| Young et al. [63] | 2016 | DMD* ‡ | H in NSG-mdx M | NF | Deletion of 725 kb, encompassing exons 45–55, restored dystrophin in muscle cells |
| Zhang et al. [23] | 2017 | DMD*† | H, mdx M | NF and MI | Used a new endonuclease (cpf1) for DMD editing in human iPSCs and mice |
| Zhu et al. [55] | 2017 | DMD*‡ | mdx M | LF 3000, AdV | Developed a fibrin gel to propagate CRISPR-cas9-corrected muscle stem cells |
* Concept proven in vitro.
† Concept proven in vivo.
‡ Concept proven ex vivo.
Abbreviations: H–human, M–mouse, mdx–mouse model of DMD, AdV–Adenovirus, AAV–Adeno-associated virus, LF–Lipofectamine, PEI–Polyethylenimine, CGBP–CRISPR-Gold nanoparticles, LF–Lipofectamine, EP–Electroporation, NF–Nucleofection, HC-AdV–High-capacity adenoviral vectors, MI–Microinjection.