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Abbreviation : scAAV9
Long Form : self-complementary adeno-associated virus 9
No. Year Title Co-occurring Abbreviation
2020 A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna. CM, CSF
2019 AAV9-mediated delivery of miR-23a reduces disease severity in Smn2B/-SMA model mice. iPSC, miRNA, SMA, SMN1
2019 Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease. CB, CSF, i.c.v
2018 Intramuscular Delivery of scAAV9-hIGF1 Prolongs Survival in the hSOD1G93A ALS Mouse Model via Upregulation of D-Amino Acid Oxidase. ALS, CNS, DAO, hIGF1
2018 Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice. SD
2018 Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain. SMA, SMN1
2016 Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3). GFP, JNCL, MeCP2
2015 Gene delivery to the spinal cord using MRI-guided focused ultrasound. BSCB, FUS, GFP, MRIgFUS
2014 Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy. CRMs, FIX, vg
10  2010 Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. SMA, SMN