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Gene Therapy

NIH, Gates launch $200 million project for HIV and sickle cell disease gene therapy

by Ryan Cross
November 2, 2019 | A version of this story appeared in Volume 97, Issue 43

The National Institutes of Health and the Bill & Melinda Gates Foundation are committing $100 million each to develop affordable gene therapies for HIV and sickle cell disease. Sickle cell could be cured with gene-replacement or gene-editing approaches, and preclinical research indicates that CRISPR gene editing could chop up HIV genomes hiding in the body. Both programs will focus on in vivo therapies, in which cells are altered inside the body. The biggest challenge for both could be lowering manufacturing costs.

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