Murtaza S. Nagree

发表

An update on gene therapy for lysosomal storage disorders

J. Medin, Murtaza S Nagree, Simone Scalia, 2019, Expert opinion on biological therapy.

rAAV-mediated over-expression of acid ceramidase prevents retinopathy in a mouse model of Farber lipogranulomatosis

J. Medin, Haoyuan Liu, Xiaoqing Pan, 2022, Gene Therapy.

Acid Ceramidase Deficiency in Mice Leads to Severe Ocular Pathology and Visual Impairment.

J. Medin, J. Carroll, A. Salmon, 2019, The American journal of pathology.

Spinal muscular atrophy-like phenotype in a mouse model of acid ceramidase deficiency

A. Pshezhetsky, M. Budde, J. Medin, 2023, Communications biology.

Towards in vivo amplification: Overcoming hurdles in the use of hematopoietic stem cells in transplantation and gene therapy.

J. Medin, Lucía López-Vásquez, Murtaza S Nagree, 2015, World journal of stem cells.

Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target

J. Medin, D. Wozniak, Yedda Li, 2019, Proceedings of the National Academy of Sciences.

Autologous, lentivirus‐modified, T‐rapa cell “micropharmacies” for lysosomal storage disorders

J. Medin, C. A. Rupar, S. R. Foley, 2022, EMBO molecular medicine.

Lipids and the cancer stemness regulatory system in acute myeloid leukemia.

Stephanie Z Xie, Murtaza S Nagree, Isabel N X Lim, 2022, Essays in biochemistry.

Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34+ Cells for Correction of Fabry Disease

A. Schambach, J. Medin, Aneal Khan, 2017, Molecular therapy. Methods & clinical development.