Base-editing comes of age and more SCD news

Alexis Komor and Nicole Gaudelli developed based editing when they were postdoc in David Liu’s laboratory at Harvard. Credit: The CRISPR Journal

The first Investigational New Drug (IND) application for base-editing technology has been cleared by the Food and Drug Administration. BEAM-101, developed by Beam Therapeutics, is an ex vivo base-editing product candidate, meaning that it uses a modified form of CRISPR capable of making single base changes without double-stranded DNA cleavage.

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Our CRISPR future, according to J. Doudna

The Nobel Prize for CRISPR is one of the most exciting ever assigned in chemistry and one of the most celebrated in the media, for reasons related to the invention and the inventors alike. On the one hand, the technique is changing the practice and the image of genetic engineering. On the other hand, Jennifer Doudna and Emmanuelle Charpentier are not merely great scientists; they are a success story in cracking the glass ceiling and a symbol of the strength of collaboration.

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Breaking or fixing? A tale of two approaches for hemoglobinopathies

Painting by Hertz Nazaire

Covid19 is affecting everyone, but it has hit the sickle cell (SCD) community particularly hard. According to STAT News the pandemic has temporarily stopped clinical trials and the introduction of new drugs, besides directly impacting SCD patients who are at high risk for severe complications from Sars-Cov2 infection and may need hospital assistance for SCD pain crises.

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