第25回ASGCT（米国遺伝子細胞治療学会）年次総会にて、当社は６件の演題について発表しました。
発表の資料は、下記リンク先からご覧ください。
https://www.dropbox.com/sh/k5uwj60yxfbqg2g/AADbwV3TAfHF0wdWZT92xAaja?dl=0

＜当社が発表した演題＞
1. MDC1A
Novel Single AAV Vector Treatment for Congenital Muscular Dystrophy Type 1A (MDC1A) Using CRISPR-GNDM® Technology
2. Vector
NGS Based Evaluation of AAV Genome Integrity for Improved Production and Function
3. Tau
Robust Suppression of Tau by CRISPR-GNDM® System for Treatment of Tauopathies
4. TTN
Utilizing CRISPR-GNDM® Mediated Gene Activation of the Extra-Large Gene Titin for the Treatment of Dilated Cardiomyopathy and Other Titinopathies
5. Angelman
Blocking SNHG14/UBE3A-ATS lncRNA Transcription with Dead Cas9 (CRISPR-GNDM®) Can Un-Silence Paternal UBE3A in an Angelman Syndrome Mouse Model
6. Immunology
Evaluation of Cas9 Mediated Immune Response Effect on Long Term Transgene Expression in WT Mice and NHPs without Immune Suppressant