Deborah A. Zygmunt

发表

rAAVrh74.MCK.GALGT2 Demonstrates Safety and Widespread Muscle Glycosylation after Intravenous Delivery in C57BL/6J Mice

B. Bolon, Ying Jia, J. Yoon, 2019, Molecular therapy. Methods & clinical development.

Short-term treatment of golden retriever muscular dystrophy (GRMD) dogs with rAAVrh74.MHCK7.GALGT2 induces muscle glycosylation and utrophin expression but has no significant effect on muscle strength

P. Nghiem, J. Kornegay, S. M. Birch, 2021, PloS one.

Active and passive immunization strategies based on the SDPM1 peptide demonstrate pre-clinical efficacy in the APPswePSEN1dE9 mouse model for Alzheimer's disease

Chiou-Miin Wang, B. Kaspar, Carlos J. Miranda, 2014, Neurobiology of Disease.

Altered MicroRNA processing in heritable pulmonary arterial hypertension: an important role for Smad-8.

S. Erzurum, S. Comhair, Lingli Wang, 2011, American journal of respiratory and critical care medicine.

Comparison of Serum rAAV Serotype-Specific Antibodies in Patients with Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Inclusion Body Myositis, or GNE Myopathy.

K. Flanigan, D. Zygmunt, K. Crowe, 2017, Human gene therapy.

Deletion of Pofut1 in Mouse Skeletal Myofibers Induces Muscle Aging-Related Phenotypes in cis and in trans

P. White, Ying Jia, Rui Xu, 2017, Molecular and Cellular Biology.

rAAVrh74.MCK.GALGT2 Protects against Loss of Hemodynamic Function in the Aging mdx Mouse Heart.

Ying Jia, Paul T Martin, Rui Xu, 2019, Molecular therapy : the journal of the American Society of Gene Therapy.

Development of Assays to Measure GNE Gene Potency and Gene Replacement in Skeletal Muscle

M. Lek, A. Lek, Patricia Lam, 2023, Journal of neuromuscular diseases.

Visualizing Muscle Sialic Acid Expression in the GNED207VTgGne-/- Cmah-/- Model of GNE Myopathy: A Comparison of Dietary and Gene Therapy Approaches.

I. Nishino, L. Rodino‐Klapac, S. Noguchi, 2021, Journal of neuromuscular diseases.

Serum Antibodies to N-Glycolylneuraminic Acid Are Elevated in Duchenne Muscular Dystrophy and Correlate with Increased Disease Pathology in Cmah-/-mdx Mice.

A. Varki, Sandra Díaz, Hai Yu, 2021, The American journal of pathology.

An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque

K. Flanigan, J. Cheatham, L. Rodino‐Klapac, 2018, Molecular therapy. Methods & clinical development.