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A primer to gene therapy: Progress, prospects, and problems

Genetic therapies based on gene addition have witnessed a variety of clinical successes and the first therapeutic products have been approved for clinical use. Moreover, innovative gene editing techniques are starting to offer new opportunities in which the mutations that underlie genetic diseases c...

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Bibliographic Details
Published in:J Inherit Metab Dis
Main Authors: Zittersteijn, Hidde A., Gonçalves, Manuel A.F.V., Hoeben, Rob C.
Format: Artigo
Language:Inglês
Published: John Wiley & Sons, Inc. 2020
Subjects:
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC7891367/
https://ncbi.nlm.nih.gov/pubmed/32510617
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jimd.12270
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