according to plos
3 Gene Editing Approaches for Sickle Cell Disease
3 Gene Editing Approaches for Sickle Cell Disease
Sickle cell disease (SCD) is a perfect candidate for gene editing.It is perhaps the best understood single-gene condition, due to a substitution of a single DNA base in the gene that encodes the beta subunit of hemoglobin, the protein that carries oxygen in the blood.Red blood cells, which bend into the sickle shape in the disease, obstructing circulation and causing excruciating pain, descend from hematopoietic ("blood-forming") stem cells (HSCs) in the bone marrow.So theoretically, a patient's stem cells can be removed, the mutation replaced in them, and then the fixed cells infused back into the patient, circumventing an immune response.
coupled with rdmag
New Gene Therapy Shows Early Signs of Alzheimer's Disease Prevention
New Gene Therapy Shows Early Signs of Alzheimer's Disease Prevention
Scientists have made some early progress in finding a potential preventative treatment for Alzheimer's disease.A team based at the Imperial College London tested a modified virus that inserted the PGC1-alpha gene into two areas of a mouse subject's brain, the hippocampus and cortex, which are susceptible to Alzheimer's disease, according to the official announcement.Previous studies performed by this team indicated the PGC1 – alpha gene played a role in preventing the formation of amyloid-beta peptide plaques.These proteins form sticky clumps that have been found in the brains of people diagnosed with this degenerative disorder.
additionally forbes
Researchers Create A Gene Therapy Treatment That May Prevent Alzheimer's Disease
Researchers Create A Gene Therapy Treatment That May Prevent Alzheimer's Disease
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