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

[HT-17-2] BBB-penetrating heteroduplex oligonucleotide

^○Tetsuya Nagata (Department of Neurology and Neurological Science, Tokyo Medical and Dental 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.

2018-Current
Research Associate Professor, Department of Neurology, Department of Neurology and Neurological Science, Tokyo Medical and Dental University
2014-2018
Research Senior Assistant Professor, Department of Neurology, Department of Neurology and Neurological Science, Tokyo Medical and Dental University
2009-2014
Section chief, Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry
2008
Assistant Professor, Department of Neurology, Okayama University School of Medicine
2005-2008
Postdoctoral fellow, Department of Neurology/Center for Motor Neuron Biology and Disease, Columbia University
2003-2005
Assistant Professor, Department of Neurology, Okayama University School of Medicine
1999
Graduated from Tohoku University Graduate School
1994
Graduated from Tohoku University School of Medicine