Google
Trusted Resources: Community Center
Online groups, photo galleries and blogs
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.
+myBinderRelated Content
-
More Curative Bone Marrow Transplants are Successful When Patients Given Double the Radiation Beforehand, Study SaysThe success rate of bone marrow transpla...
-
Safety and early hints of benefit seen in phase 1b trial of PF-04447943Sickle cell disease (SCD) patients were ...
-
Gamida Cell Announces $3.5 Million Grant from the Israeli GovernmentGamida Cell, a leader in cellular and im...
-
A Multicenter Phase II Trial of Unrelated Donor Reduced Intensity Bone Marrow Transplantation for Children with Seve...Background: Children with severe sickle...
-
SCDAA Statement About Gene Therapy ApprovalOn Dec. 8, 2023, the Food and Drug Admin...
-
Greenville program tries team approach to improve lifespan of sickle cell disease patientsDr. Alan Anderson who was involved with ...
-
Podcast: How New Gene Therapies for SCD May Impact Women’s FertilityAs we closed 2023, the Food and Drug Ad...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
