BC Extra | Dec 11, 2019
Tools & Techniques

Editas shows better gene editing using Cas9 alternative for sickle cell, thalassemia

Editas is using an alternative to Cas9 to develop a differentiated sickle cell and β thalassemia CRISPR gene therapy. Preclinical data presented Monday at ASH suggest that ex vivo gene editing could be more effective...
BC Innovations | Mar 20, 2019
Translation in Brief

Sangamo’s ZFN linkers finger new options

Sangamo is using new linkers to expand the structural variety of the ZFNs it designs and increase the odds of finding ZFNs with high editing efficiency and specificity for their targets. Conventional zinc finger nucleases...
BC Innovations | Dec 8, 2017
Targets & Mechanisms

Epiphenomena at ASH

While it’s no surprise that cell therapy research is dominant in hematology, the emergence of epigenetics as an equally large focus at this year’s ASH meeting is more notable, reinforcing the resurgence of a field...
BC Innovations | Nov 17, 2016
Translation in Brief

Editing hemoglobin

Three independent teams have demonstrated three different ways of using CRISPR to treat sickle cell disease, paving the way for correcting genetic mutations in a disease where the only long-term therapeutic option is blood transplants...
BC Innovations | Sep 22, 2016
Distillery Therapeutics

Therapeutics: Hemoglobin subunit γ 1 (HBG1); HBG2; CRISPR-associated protein 9 (Cas9)

Hematology INDICATION: Sickle cell disease (SCD) Patient sample and cell culture studies suggest enhancing fetal hemoglobin production in hematopoietic stem and progenitor cells (HSPCs) using CRISPR-based gene editing could help treat SCD. In HSPCs isolated...
BC Innovations | Feb 28, 2013
Distillery Therapeutics

Indication: Hematology

Indication Target/marker/pathway Summary Licensing status Publication and contact information Hematology Thalassemia; sickle cell disease Lysine-specific histone demethylase 1 (KDM1A; LSD1) In vitro and mouse studies suggest inhibiting LSD1 could help treat sickle cell disease and...
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