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Trusted Resources: News & Events
Latest announcements and gatherings
The FDA granted fast track designation to CTX001, a gene-edited hematopoietic stem cell therapy, for the treatment of sickle cell disease.
CTX001 (CRISPR Therapeutics, Vertex Pharmaceuticals Inc.) is an investigational, autologous gene therapy under evaluation for patients with severe hemoglobinopathies. The ex-vivo therapy involves engineering a patient’s hematopoietic stem cells to produce high levels of fetal hemoglobin in red blood cells, with the goal of alleviating transfusion requirements for patients with beta-thalassemia and sickle crises for patients with sickle cell disease.
news & eventsSickle Cell Patient Receives CRISPR Gene TherapyMany human diseases can be traced back t...
news & events1st Patients to Get CRISPR Gene-Editing Treatment Continue to ThriveThe last thing a lot of people want to d...
news & eventsProgress in pursuit of sickle cell cureBao, in collaboration with Baylor Colleg...
news & eventsSolution to 50-year-old mystery could lead to gene therapy for common blood disordersIn a landmark study that could lead to n...
news & eventsMaxCyte, Inc. to Present Positive Preclinical Data for Sickle Cell DiseaseMaxCyte, a US-based global company dedic...
videos & visualsJennifer Doudna on ethics of gene editinghttps://www.youtube.com/watch?v=8Ijr1ccY...
Community CenterSickle Cell Disease: Gene-Editing Tools Point to Possible Ultimate CureRecent advances in CRISPR/Cas9 gene-edit...
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