Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy von Fu, Haiyan, Meadows, Aaron S, Pineda, Ricardo J, Kunkler, Krista L, Truxal, Kristen V, McBride, Kim L, Flanigan, Kevin M, McCarty, Douglas M

Volltext

Systemic gene transfer of scAAV9.U1a.hSGSH for MPS IIIA: tolerability and preliminary evidence for a biochemical effect von Flanigan, Kevin M., Truxal, Kristen V., McBride, Kim L., McNally, Kelly A., Kunkler, Krista L., Zumberge, Nicholas A., Martin, Lisa, Aylward, Shawn C., Corridore, Marco, McKee, Christopher, Fu, Haiyan, McCarty, Douglas M.

Volltext

Differential prevalence of antibodies against adeno-associated virus in healthy children and patients with mucopolysaccharidosis III: perspective for AAV-mediated gene therapy von Fu, Haiyan, Meadows, Aaron S, Pineda, Ricardo J, Kunkler, Krista L, Truxal, Kristen V, McBride, Kim L, Flanigan, Kevin, McCarty, Douglas M

Volltext

Perception of Object Length by Sound von Carello, Claudia, Anderson, Krista L., Kunkler-Peck, Andrew J.

Volltext

Prospective natural history study of mucopolysaccharidosis types IIIA and IIIB (Sanfilippo syndrome) von Truxal, Kristen, McBride, Kim L., McNally, Kelly, Kunkler, Krista, Twersky, Jill, Grimes, Rebecca, Sherrod-Canaan, Tamar, Aylward, Shawn, Alfano, Lindsay, Berry, Katherine, Lowes, Linda, Zumberge, Nicholas, Martin, Lisa, Corridore, Marco, McKee, Christopher, McCarty, Douglas, Fu, Haiyan, Flanigan, Kevin M.

Volltext

Design of a phase I/II gene transfer clinical trial of rAAV9.CMV.hNAGLU for mucopolysaccharidosis type IIIB von McBride, Kim L., Truxal, Kristen, McNally, Kelly, Kunkler, Krista, Sherrod-Canaan, Tamar, Aylward, Shawn, Miller, Tim, Zumberge, Nicholas, Martin, Lisa, Corridore, Marco, McKee, Christopher, McCarty, Douglas, Fu, Haiyan, Flanigan, Kevin

Volltext