"We'd like to say, 'Yes,'" he said, "but we don't really know yet.That's something we hope to learn from an eventual clinical trial." Scientists have discovered a gene-editing technology that could efficiently and accurately correct the genetic defects that underlie certain diseases, positioning the new tool as the basis for the ...
Scientists have successfully used gene editing to repair 20 to 40 percent of stem and progenitor cells taken from the peripheral blood of patients with sickle cell disease, according to Rice University bioengineer Gang Bao.
Bao, in collaboration with Baylor College of Medicine, Texas Children's Hospital and Stanford University, is working to find a cure for the hereditary disease.
The gene-edited cells were injected into the bone marrow of immunodeficient mice and tested after 19 weeks to see how many retained the edit.
"The rate of repair remained stable, which is great," Bao said.
[tags: Red blood cell, Sickle-cell disease, Hemoglobin] - Sickle cell anemia (SCA) is one of the most common inherited genetic diseases.
It is frequently found in tropical regions of the world.[tags: Sickle-cell disease, Red blood cell, Sickle] - sickle cell anemia sickle cell anemia (sickle cell disease) is a disorder of the blood caused by an inherited abnormal hemoglobin (the oxygen-carrying protein within the red bloodcells).The abnormal hemoglobin causes distorted (sickled) red blood cells.It primary affects people from African descend, followed by people of Arab, Indian and Hispanic descent.SCA changes the conformation of hemoglobin, the globular protein inside red blood cells that helps transport oxygen throughout the body.A single DNA mutation causes the body to make sticky, crescent-shaped red blood cells that contain abnormal hemoglobin and can block blood flow in limbs and organs.In his talk at the annual American Association for the Advancement of Science meeting in Austin today, Bao revealed results from a series of tests to see whether CRISPR/Cas9-based editing can fix the mutation.Those will then be healthy blood cells." Bao's lab collaborated with Vivien Sheehan, an assistant professor of pediatrics and hematology at Baylor and a member of the sickle cell program at Texas Children's, to collect stem and progenitor cells (CD34-positive cells) from patients with the disease.These were then edited in the Bao lab with CRISPR/Cas9 together with a custom template, a piece of DNA designed to correct the mutation.Thankfully, without a cure, we are able to lengthen the lives of the individuals suffering from sickle cell disease with treatment....[tags: Red blood cell, Hemoglobin, Sickle-cell disease] - Sickle cell anemia is an inherited pathosis that must have two copies of an aberrant gene present in order for the condition to cultivate and then, transcribe the inadequate hemoglobin molecule (Ricci, Kyle, & Carman, 2013).