scholarly journals 372. Prevalence of Anti-AAV8 Neutralizing Antibodies and ARSB Cross-Reactive Immunologic Material in MPS VI Patients Candidates for a Gene Therapy Trial

2015 ◽  
Vol 23 ◽  
pp. S148
Author(s):  
Rita Ferla ◽  
Pamela Claudiani ◽  
Marco Savarese ◽  
Karen Kozarsky ◽  
Rossella Parini ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Neutralizing Antibodies ◽  
Gene Therapy Trial ◽  
Mps Vi ◽  
Therapy Trial

ASPIRO Gene Therapy Trial in X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Efficacy and Safety Findings

2021 ◽  
Author(s):  
A. Blaschek ◽  
P. B. Shieh ◽  
N. Kuntz ◽  
J. J. Dowling ◽  
W. Müller-Felber ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Efficacy And Safety ◽  
Myotubular Myopathy ◽  
Gene Therapy Trial ◽  
Therapy Trial
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