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Gene Therapy Using Stem Cells

Viral-mediated gene augmentation therapy has recently shown success in restoring vision to patients with retinal degenerative disorders. Key to this success was the availability of animal models that accurately presented the human phenotype to test preclinical efficacy and safety. These exciting stu...

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Détails bibliographiques
Publié dans:Cold Spring Harb Perspect Med
Auteurs principaux: Burnight, Erin R., Wiley, Luke A., Mullins, Robert F., Stone, Edwin M., Tucker, Budd A.
Format: Artigo
Langue:Inglês
Publié: Cold Spring Harbor Laboratory Press 2015
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4382729/
https://ncbi.nlm.nih.gov/pubmed/25395375
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1101/cshperspect.a017434
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