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Sequence Replacement to Cure Sickle Cell Disease
Next-generation gene editing is already transforming the way scientists do research, but it also holds a great deal of promise for the cure of genetic diseases. One of the most tractable genetic diseases for gene editing is sickle cell disease (SCD). The molecular basis has been known since 1949, so it’s relatively well understood. Its root cause is in bone marrow stem cells (aka hematopoietic stem cells, or “HSCs”), which are easy to get to with editing reagents. It’s monogenic and recessive, so you only need to reverse one disease allele for a cure.
videos & visualsBlood Transfusions: How, Why, and When? (Part 1)https://www.youtube.com/watch?v=KRjjp0Jw...
education & researchThe Experience and Health-Related Quality of Life After Haploidentical Stem Cell Transplantation for Adults With Sic...Haploidentical hematopoietic stem cell t...
education & researchStroke Avoidance for Children in REpública Dominicana (SACRED): Protocol for a Prospective Study of Stroke Risk and...Background: In the Dominican Republic,...
news & eventsFDA Clears Graphite Bio to Begin Trial for Gene Therapy in Sickle Cell DiseaseIn response to Graphite Bio’s investig...
news & eventsSickle-Cell Patients See Hope in CRISPRSickle-cell disease is one of the most c...
news & eventsIn A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic DisorderFor the first time, doctors in the U.S. ...
news & eventsNew Guidelines for Managing Sickle Cell Disease Presented at ACP Internal Medicine MeetingNew guidelines for therapeutic strategie...
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