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2022 CCDS Symposium Session 5: 'Gene Therapy Approaches for CCDS'
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Gabriella Fernandes-Pires - Lausanne University Hospital
"AAV strategy to treat creatine transporter deficiency in the Slc6a8Y389C/y KI rat"
Laura Baroncelli - Neuroscience Institute CNR
"Creatine transporter deficiency: The long journey to successful therapy"
Gerald Lipshutz - UCLA
"AAV-based gene therapy restores cerebral creatine, reduces guanidinoacetate, and resolves of behavioral abnormalities in a mouse model of GAMT deficiency"
Q&A Panel with Lara Duran-Trio, Gabriella Fernandes-Pires, Laura Baroncelli, and Gerald Lipshutz (Moderator: Andreas Schulze)
#Creatinedeficiencies #CCDS #Creatineinfo #CCDS2022
"AAV strategy to treat creatine transporter deficiency in the Slc6a8Y389C/y KI rat"
Laura Baroncelli - Neuroscience Institute CNR
"Creatine transporter deficiency: The long journey to successful therapy"
Gerald Lipshutz - UCLA
"AAV-based gene therapy restores cerebral creatine, reduces guanidinoacetate, and resolves of behavioral abnormalities in a mouse model of GAMT deficiency"
Q&A Panel with Lara Duran-Trio, Gabriella Fernandes-Pires, Laura Baroncelli, and Gerald Lipshutz (Moderator: Andreas Schulze)
#Creatinedeficiencies #CCDS #Creatineinfo #CCDS2022
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