[HT-17] Hot Topics 17
Update of Oligonucleotide Therapy for CNS diseases；From Basic to Clinic

[HT-17-4] Basic biology and clinical application of ASO to SMA

^○Kentaro Sahashi (Department of Neurology, Nagoya University, Japan)

Following antisense oligonucleotide, nusinersen/Spinraza for SMA, first siRNA drug, givosiran/Fitusiran for TTR amyloidosis (familial amyloid neuropathy) has been approved in Japan, and SOD1 and C9ORF72 for ALS, tau for Alzheimer disease, PSP/CBD, synuclein for Parkinson disease, DLB and MSA, and Huntinting for Huntington disease are also started for clinical stages. This rapid and splendid progress of oligonucleotide therapy are now a main stream for development of DMT to neurological diseases. In this symposium, basic chemistry, delivery and biology of oligonucleotide drug are reviewed, and current clinical status for approved oligonucleotide drugs for neurological diseases are to be updated.

1999 Nagoya Univ. School of Medicine. M.D.
2008 Nagoya Univ. Grad. School of Medicine. Ph.D.

1999 Residency in Japanese Red Cross Nagoya First Hospital
2004 Graduate course in Department of Cell Information Medicine, Nagoya Univ. Grad. School of Medicine
2008 Postdoctoral fellow in Cold Spring Harbor Laboratory
2013 Staff in Department of Neurology, Nagoya Univ. Grad. School of Medicine
2016 Assistant professor in Nagoya Univ. Hospital