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Papers of the Week


Papers: 6 Aug 2022 - 12 Aug 2022


2022 Aug 04


Trends Genet

Effective therapies for sickle cell disease: are we there yet?

Authors

Crossley M, Christakopoulos GE, Weiss MJ
Trends Genet. 2022 Aug 04.
PMID: 35934593.

Abstract

Sickle cell disease (SCD) is a common genetic blood disorder associated with acute and chronic pain, progressive multiorgan damage, and early mortality. Recent advances in technologies to manipulate the human genome, a century of research and the development of techniques enabling the isolation, efficient genetic modification, and reimplantation of autologous patient hematopoietic stem cells (HSCs), mean that curing most patients with SCD could soon be a reality in wealthy countries. In parallel, ongoing research is pursuing more facile treatments, such as in-vivo-delivered genetic therapies and new drugs that can eventually be administered in low- and middle-income countries where most SCD patients reside.