Under the hood: The molecular biology driving gene therapy for the treatment of sickle cell disease
Transfusion and Apheresis Science, ISSN: 1473-0502, Vol: 61, Issue: 5, Page: 103566
2022
- 23Captures
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Example: if you select the 1-year option for an article published in 2019 and a metric category shows 90%, that means that the article or review is performing better than 90% of the other articles/reviews published in that journal in 2019. If you select the 3-year option for the same article published in 2019 and the metric category shows 90%, that means that the article or review is performing better than 90% of the other articles/reviews published in that journal in 2019, 2018 and 2017.
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Example: if you select the 1-year option for an article published in 2019 and a metric category shows 90%, that means that the article or review is performing better than 90% of the other articles/reviews published in that journal in 2019. If you select the 3-year option for the same article published in 2019 and the metric category shows 90%, that means that the article or review is performing better than 90% of the other articles/reviews published in that journal in 2019, 2018 and 2017.
Citation Benchmarking is provided by Scopus and SciVal and is different from the metrics context provided by PlumX Metrics.
Metrics Details
- Captures23
- Readers23
- 23
Article Description
Gene therapy will soon become the dominant modality for treating of sickle cell disease (SCD). Currently, three technologies are the most promising: expression of transgenic globin genes via a lentiviral vector, controlled mutation of the β-globin control cluster by transgenic CRISPR-based ribonucleoprotein, and suppression of BCL11a mRNA by shRNA. In this review, we discuss the mechanism of each technology and how they correct the SCD pathology at the molecular level. We conclude by discussing potential directions future therapy may take.
Bibliographic Details
http://www.sciencedirect.com/science/article/pii/S147305022200252X; http://dx.doi.org/10.1016/j.transci.2022.103566; http://www.scopus.com/inward/record.url?partnerID=HzOxMe3b&scp=85138108697&origin=inward; http://www.ncbi.nlm.nih.gov/pubmed/36115765; https://linkinghub.elsevier.com/retrieve/pii/S147305022200252X; https://dx.doi.org/10.1016/j.transci.2022.103566
Elsevier BV
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