Stanford researchers have developed a genetic strategy using antisense oligonucleotides (ASO) to reduce the levels of PCDH19 in human forebrain neurons, as a therapeutic approach for PCDH19-related encephalopathy.
Scientists in Sergiu Pasca's group at Stanford University have used patient-derived organoids, assembloids and in vivo transplantation to discover and validate an antisense oligonucleotide drug for the treatment of Timothy syndrome.