At the 25th Annual Meeting of the The American Society of Gene & Cell Therapy (ASGCT), we made presentations on 6 items.
Please refer to the link below for the presentation materials.

<Announced items>
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