Neuron-derived hiPSC: an in vitro model for the development of a gene therapy for myotonic dystrophy type 1

0

Myotonic dystrophy type 1 (DM1) is a multisystemic disease with dominant transmission that affects not only muscle but also many organs such as the brain. Cerebral damage included psychomotor disorder, mental retardation, loss of visuospatial and memory functions. Antisense oligonucleotides (AOs) strategy for the development of a gene therapy for DM1 brain deficits is limited by the fact that AOs do not cross the blood-brain barrier following systemic administration indicating that other delivery methods will have to be considered.

Read More...

Leave A Reply