scholarly journals Prevalence of Anti–Adeno-Associated Virus Serotype 8 Neutralizing Antibodies and Arylsulfatase B Cross-Reactive Immunologic Material in Mucopolysaccharidosis VI Patient Candidates for a Gene Therapy Trial

2015 ◽  
Vol 26 (3) ◽  
pp. 145-152 ◽  
Author(s):  
Rita Ferla ◽  
Pamela Claudiani ◽  
Marco Savarese ◽  
Karen Kozarsky ◽  
Rossella Parini ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Neutralizing Antibodies ◽  
Adeno Associated Virus ◽  
Gene Therapy Trial ◽  
Arylsulfatase B ◽  
Virus Serotype ◽  
Mucopolysaccharidosis Vi ◽  
Therapy Trial

Phase 2–3 gene therapy trial using adeno-associated virus vector for patients with mucopolysaccharidosis type IIIA

2020 ◽  
Vol 129 (2) ◽  
pp. S162
Author(s):  
Frits Wijburg ◽  
Kristian Aquilina ◽  
Bénédicte Heron ◽  
Anupam Chakrapani ◽  
Ron Crystal ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Virus Vector ◽  
Mucopolysaccharidosis Type ◽  
Phase 2 ◽  
Adeno Associated Virus ◽  
Gene Therapy Trial ◽  
Type Iiia ◽  
Therapy Trial ◽  
Mucopolysaccharidosis Type Iiia

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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