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Towards accessible gene therapy: in vivo base editing to cure Sickle Cell Disease
From the Cancer Consortium Lieber Lab, Department of Laboratory Medicine and Pathology, University of Washington
Advocating for bringing gene therapy to low-income countries
From the Adair Lab, Translational Science and Therapeutics Division
Hitting ‘undo’ to cure Sickle Cell Disease
From the Lieber and Kiem Labs, Hematologic Malignancies Program, Cancer Consortium.
Advances in blood disorders research continue despite COVID-19
Expanding use of CAR T-cell therapies, gene editing lead to insights at virtual annual meeting of the American Society of Hematology
$3.5M grant to develop safer treatment for inherited blood disorders
Scientists will study radioactive particles that can precisely deliver a potent punch
A look at emerging sickle cell disease therapies
Teams at the University of Washington School of Pharmacy and Fred Hutch will model the clinical and economic burden of sickle cell disease and the potential benefits of emerging therapies
Targeting a subset of stem cells shows lasting, therapeutically relevant gene editing in blood cells
A new Science Translational Medicine paper is first to report how editing a portion of stem cells with CRISPR/Cas9 is sufficient for long-term reactivation of therapeutic hemoglobin
Gene editing shows promise for sickle cell and related disorders
Preclinical results offer new hope in group of dangerous inborn diseases
Gene therapy for sickle cell disease, and more
$3.7 million grant to fund novel gene therapy approach for dangerous genetic disorders of hemoglobin
What's new in blood diseases
Four things to watch from the 2016 annual meeting of the American Society of Hematology