The Potential of Induced Pluripotent Stem Cells to Test Gene Therapy Approaches for Neuromuscular and Motor Neuron Disorders

The reprogramming of somatic cells into induced pluripotent stem cells (iPSCs) represents a major advance for the development of human disease models. The emerging of this technique fostered the concept of “disease in a dish,” which consists into the generation of patient-specific models in vitro. C...

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Bibliographic Details
Main Authors: Marisa Cappella, Sahar Elouej, Maria Grazia Biferi
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-04-01
Series:Frontiers in Cell and Developmental Biology
Subjects:
AAV
NMD
MND
Online Access:https://www.frontiersin.org/articles/10.3389/fcell.2021.662837/full