Gene Targets for in Utero Therapy in Down Syndrome: Potentials and Pitfalls

January 16, 2017

(News-Medical) – Sequencing of HSA21 revealed the presence of 364-protein coding genes on this chromosome. Fortunately, HSA21 is the smallest and among the poorest chromosomes in terms of the number of functional genes. Among the >300 genes, only a small number are expressed in the brain regions associated with learning and memory and have been linked to normal or abnormal functions in the nervous system. Normalizing the expression of a number of critical genes would be able to prevent a part of nervous system abnormalities in DS.