Gene of the Month – August: TTR
Permanent and efficient inactivation of the TTR gene by genome editing is the aim of a novel therapeutic approach currently being investigated for the treatment of a rare congenital disease, [...]
Newly identified biallelic variants in MFSD2A expand clinical spectrum of a very rare microcephaly-associated disease
Researchers of the Institute of Human Genetics at the University Medical Center Göttingen, together with their colleagues at the Institute of Human Genetics in Essen and other collaboration [...]