Publications by authors named "Todd D Borland"
The only cure of HIV has been achieved in a small number of people who received a hematopoietic stem cell transplant (HSCT) comprising allogeneic cells carrying a rare, naturally occurring, homozygous deletion in the CCR5 gene. The rarity of the mutation and the significant morbidity and mortality of such allogeneic transplants precludes widespread adoption of this HIV cure. Here, we show the application of CRISPR/Cas9 to achieve >90% CCR5 editing in human, mobilized hematopoietic stem progenitor cells (HSPC), resulting in a transplant that undergoes normal hematopoiesis, produces CCR5 null T cells, and renders xenograft mice refractory to HIV infection.
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- ATTR amyloidosis is a serious and often deadly disease caused by a protein called transthyretin (TTR) building up in the body.
- Scientists are exploring a new treatment method called RNA interference (RNAi), which can help reduce the amount of TTR made in the liver.
- In tests with mice, using RNAi significantly helped to lower TTR levels and even shrank existing TTR buildups more effectively than a current treatment called tafamidis.