scholarly journals 114. AAV-Mediated Gene Therapy Reduces Glycosphingolipid Storage in Adult GM1 Gangliosidosis Mice

2008 ◽  
Vol 16 ◽  
pp. S44
Keyword(s):  
Gene Therapy ◽  
Gm1 Gangliosidosis

scholarly journals Novel Biomarkers of Human GM1 Gangliosidosis Reflect the Clinical Efficacy of Gene Therapy in a Feline Model

2017 ◽  
Vol 25 (4) ◽  
pp. 892-903 ◽  
Author(s):  
Heather L. Gray-Edwards ◽  
Debra S. Regier ◽  
Jamie L. Shirley ◽  
Ashley N. Randle ◽  
Nouha Salibi ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Clinical Efficacy ◽  
Gm1 Gangliosidosis ◽  
Novel Biomarkers ◽  
Feline Model

scholarly journals 373. Optimization of AAV-Gene Therapy for GM1-Gangliosidosis

2016 ◽  
Vol 24 ◽  
pp. S149 ◽  
Author(s):  
Lorelei Stoica ◽  
Sophia Todeasa ◽  
Jaclyn Lata ◽  
Douglas Martin ◽  
Miguel Sena-Esteves
Keyword(s):  
Gene Therapy ◽  
Gm1 Gangliosidosis

scholarly journals 605. Cerebrospinal Fluid for Delivery of AAV Gene Therapy in GM1 Gangliosidosis

2016 ◽  
Vol 24 ◽  
pp. S240
Author(s):  
Amanda L. Gross ◽  
Heather Gray-Edwards ◽  
Bryan Murdock ◽  
Amanda Taylor ◽  
Brandon Brunson ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Cerebrospinal Fluid ◽  
Gm1 Gangliosidosis

scholarly journals 361. IV Gene Therapy Corrects Feline GM1 Gangliosidosis Long Term

2016 ◽  
Vol 24 ◽  
pp. S144-S145
Author(s):  
Heather Edwards ◽  
Ashley Randle ◽  
Miguel Sena-Esteves ◽  
Douglas Martin
Keyword(s):  
Gene Therapy ◽  
Gm1 Gangliosidosis
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