Journal Article Review Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't Ppublish 10 citations
PubMed: 35934593 DOI: 10.1016/j.tig.2022.07.003
Effective therapies for sickle cell disease: are we there yet?
Trends Genet 38(12):1284-1298
Authors: Merlin Crossley, Georgios E Christakopoulos, Mitchell J Weiss
Affiliations
- School of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, Australia 2052.
- Department of Oncology, St Jude Children's Research Hospital, Memphis, TN 38105, USA.
- Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
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.
MeSH
Anemia, Sickle Cell, Genetics, Clustered Regularly Interspaced Short Palindromic Repeats, Cells, Therapeutics, Research