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Genome Editing for β-Hemoglobinopathies: Advances and Challenges

β-hemoglobinopathies are the most common genetic disorders worldwide and are caused by mutations affecting the production or the structure of adult hemoglobin. Patients affected by these diseases suffer from anemia, impaired oxygen delivery to tissues, and multi-organ damage. In the absence of a com...

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書目詳細資料
發表在:J Clin Med
Main Authors: Frati, Giacomo, Miccio, Annarita
格式: Artigo
語言:Inglês
出版: MDPI 2021
主題:
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC7865242/
https://ncbi.nlm.nih.gov/pubmed/33525591
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jcm10030482
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