About: Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells

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About: Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells Goto Sponge NotDistinct Permalink

An Entity of Type : schema:ScholarlyArticle, within Data Space : covidontheweb.inria.fr associated with source document(s)

Attributes	Values
type	Academic Article research paper schema:ScholarlyArticle
isDefinedBy	Covid-on-the-Web dataset
has title	Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells
Creator	Bertoni, Carmen Hermano, Carlos Pinheiro, J Puri, Pier Tedesco, Francesco
Source	PMC
abstract	The progressive loss of muscle mass characteristic of many muscular dystrophies impairs the efficacy of most of the gene and molecular therapies currently being pursued for the treatment of those disorders. It is becoming increasingly evident that a therapeutic application, to be effective, needs to target not only mature myofibers, but also muscle progenitors cells or muscle stem cells able to form new muscle tissue and to restore myofibers lost as the result of the diseases or during normal homeostasis so as to guarantee effective and lost lasting effects. Correction of the genetic defect using oligodeoxynucleotides (ODNs) or engineered nucleases holds great potential for the treatment of many of the musculoskeletal disorders. The encouraging results obtained by studying in vitro systems and model organisms have set the groundwork for what is likely to become an emerging field in the area of molecular and regenerative medicine. Furthermore, the ability to isolate and expand from patients various types of muscle progenitor cells capable of committing to the myogenic lineage provides the opportunity to establish cell lines that can be used for transplantation following ex vivo manipulation and expansion. The purpose of this article is to provide a perspective on approaches aimed at correcting the genetic defect using gene editing strategies and currently under development for the treatment of Duchenne muscular dystrophy (DMD), the most sever of the neuromuscular disorders. Emphasis will be placed on describing the potential of using the patient own stem cell as source of transplantation and the challenges that gene editing technologies face in the field of regenerative biology.
has issue date	2014-04-21(xsd:dateTime)
bibo:doi	10.3389/fphys.2014.00148
bibo:pmid	24795643
has license	cc-by
sha1sum (hex)	1f7f414d82475a8c5b2272163e6f31b474904486
schema:url	https://doi.org/10.3389/fphys.2014.00148
resource representing a document's title	Emerging gene editing strategies for Duchenne muscular dystrophy targeting stem cells
has PubMed Central identifier	PMC4001063
has PubMed identifier	24795643
schema:publication	Front Physiol
resource representing a document's body	covid:1f7f414d82475a8c5b2272163e6f31b474904486#body_text
is schema:about of	named entity 'DUCHENNE MUSCULAR DYSTROPHY' named entity 'GENE EDITING' named entity 'TARGETING' named entity 'STRATEGIES' named entity 'STEM CELLS' named entity 'genomic DNA' named entity 'adenoviral vectors' named entity 'cell-mediated' named entity 'Gene editing' named entity 'motor neurons' named entity 'plasmid vectors' named entity 'transfected' named entity 'fluorescence' named entity 'nucleases' named entity 'gene' named entity 'motor neuron' named entity 'genetically modified cells' named entity 'CD133' named entity 'mice' named entity 'muscular dystrophies' named entity 'secondary structure' named entity 'deletions' named entity 'genetic defect' named entity 'stem cells' named entity 'PNAs' named entity 'mRNA' named entity 'toxicity' named entity 'myogenic' named entity 'motor neuron' named entity 'single point mutation' named entity 'dystrophin' named entity 'autosomal recessive' named entity 'nucleases' named entity 'stem cells' named entity 'dystrophin' named entity 'replication fork' named entity 'animal models' named entity 'CpG' named entity 'stem cells' named entity 'nucleases' named entity 'nucleases' named entity 'MyoD' named entity 'stem cells' named entity 'iPSCs' named entity 'FIGURE 1' named entity 'mRNA' named entity 'stem cells' named entity 'chimeric' named entity 'HIV' named entity 'Clinical trials' named entity 'gene' named entity 'base pair' named entity 'SMN1' named entity 'lentiviral vectors' named entity 'nucleases' named entity 'skeletal muscles' named entity 'chimeric DNA' named entity 'stem cells' named entity 'protein' named entity 'gene editing' named entity 'nucleases' named entity 'dystrophin' named entity 'double-strand break' named entity 'stem cells' named entity 'transversion' named entity 'DNA-binding domains' named entity 'stem cells' named entity 'dystrophin' named entity 'exon' named entity 'gene' named entity 'epithelial cell' named entity 'peptide' named entity 'immune rejection' named entity 'DNA-binding domain' named entity 'gene editing' named entity 'gene' named entity 'genome'

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