A. McVie‐Wylie

发表

Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle.

Songtao Li, D. Bali, D. Koeberl, 2011, Molecular genetics and metabolism.

Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.

D. Koeberl, D. K. Benjamin, Baodong Sun, 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

Biochemical and pharmacological characterization of different recombinant acid alpha-glucosidase preparations evaluated for the treatment of Pompe disease.

M. O'Callaghan, N. Raben, J. McPherson, 2008, Molecular genetics and metabolism.

An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe disease

S. Packman, O. Abdul-Rahman, P. Tanpaiboon, 2018, Genetics in Medicine.

Molecular cloning of a novel member of the GLUT family of transporters, SLC2a10 (GLUT10), localized on chromosome 20q13.1: a candidate gene for NIDDM susceptibility.

Yuan-Tsong Chen, A. McVie-Wylie, D. R. Lamson, 2001, Genomics.

Recombinant human acid α-glucosidase enzyme therapy for infantile glycogen storage disease type II: Results of a phase I/II clinical trial

J. Charrow, J. Phillips, Yuan-Tsong Chen, 2001, Genetics in Medicine.

Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase.

N. Raben, A. Amalfitano, P. Plotz, 1999, Proceedings of the National Academy of Sciences of the United States of America.

Long-term efficacy after [E1-, polymerase-] adenovirus-mediated transfer of human acid-alpha-glucosidase gene into glycogen storage disease type II knockout mice.

A. Amalfitano, Y. T. Chen, A. McVie-Wylie, 2001, Human gene therapy.

Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice.

N. Raben, R. Mattaliano, Seng H. Cheng, 2005, The Biochemical journal.

Development of a simple and rapid method for producing non‐fucosylated oligomannose containing antibodies with increased effector function

Scott Estes, Qun Zhou, S. Estes, 2008, Biotechnology and bioengineering.

Transcriptional response to GAA deficiency (Pompe disease) in infantile-onset patients.

K. Klinger, S. Marie, S. Madden, 2012, Molecular genetics and metabolism.

Tissue-specific inactivation of murine M6P/IGF2R.

R. Waterland, R. Jirtle, T. Orton, 2003, The American journal of pathology.

Inhibiting TGF-β activity improves respiratory function in mdx mice.

B. Wentworth, Seng H. Cheng, Kelly A Keefe, 2011, The American journal of pathology.

The influence of a polymorphism in the gene encoding angiotensin converting enzyme (ACE) on treatment outcomes in late-onset Pompe patients receiving alglucosidase alfa

Xiwen Ma, R. Pomponio, A. McVie-Wylie, 2016, Molecular genetics and metabolism reports.

Multiple muscles in the AMD quail can be “cross‐corrected” of pathologic glycogen accumulation after intravenous injection of an [E1‐, polymerase‐] adenovirus vector encoding human acid‐α‐glucosidase

A. Amalfitano, T. Kikuchi, M. Mizutani, 2003, The journal of gene medicine.

Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis

S. Boye, S. Boye, M. Lukason, 2022, Molecular therapy. Methods & clinical development.

GZ402668, a Next-Generation Anti-CD52 Antibody, Displays Decreased Proinflammatory Cytokine Release In Vitro (P3.068)

V. Dhawan, R. Sendak, J. Kaplan, 2016 .

Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial.

A. McVie-Wylie, A. McVie‐Wylie, Y. T. Chen, 2001, Genetics in medicine : official journal of the American College of Medical Genetics.