Using a patient's own stem cells, researchers have corrected the genetic alteration that causes sickle cell disease, a painful, disabling inherited blood disorder that affects mostly African-Americans. The corrected stem cells were coaxed into immature red blood cells in a test tube that then turned on a normal version of the gene
Research will make the study of diseases such as Parkinson's and Alzheimer's easier, and could lead to personalized therapies for a variety of neurodegenerative disorders. The nerve cells generated by this new method show the same functional characteristics as the mature cells found in the body, making them much better models for the study of age-related diseases such as Parkinson's and Alzheimer's, and for the testing of new drugs.
In the August 1 issue of CELL, researchers from the Gene and Stem Cell Therapy Program at Sydney's Centenary Institute revealed another function of introns, or noncoding nucleotide sequences, in DNA. They reported that gene-sequencing techniques and computer analysis allowed them to demonstrate how granulocytes use noncoding DNA to regulate the activity of a group of genes that determines the cells' shape and function.
A new study shows that gene editing using CRISPR/Cas9 technology can work in rhesus monkey embryos.
The results, published in the current issue of Human Molecular Genetics, open the door for pursuing gene editing in nonhuman primates as models for new therapies, including pharmacological, gene-, and stem cell-based therapies, says Keith Latham, animal science professor at Michigan State University and lead author of the study.