University System of Maryland

Icahn School of Medicine at Mount Sinai

University of Pennsylvania

Silvia Muro Galindo

Edward Howard Schuchman

Vladimir R Muzykantov

Rasa Ghaffarian

Ming Meng

Carmen Garnacho Montero

The present invention relates to compositions and methods for delivering lysosomal proteins. The compositions and methods described herein permit the targeted delivery of exogenous lysosomal proteins to cell surface proteins that allow their internalization via non-clathrin pathways. The present invention further relates to the use of the compositions and methods for enzyme replacement therapy of lysosomal storage diseases. Nucleic acids, recombinant cells and kits useful for making and using the compositions of the invention are also provided.

Targeted protein replacement for the treatment of lysosomal storage disorders

A system and method for transcellular transport of compositions containing agents (e.g., research, analytical, reporter or molecular probes, diagnostic and therapeutic agents, biologically active agents, research agents, analytical agents, imaging agents, monitoring agents, enzymes, proteins, peptides, nucleic acids, lipids, sugars, hormones, lipoproteins, chemicals, viruses, bacteria, cells, including modified cells, biosensors, markers, antibodies and/or ligands) across the gastrointestinal epithelial layer including use of a composition containing the agent and a targeting moiety, specific for a determinant at the target location. An exemplary composition of the system includes an anti-ICAM antibody targeting moiety, specific for targeting ICAM-1. The system enables effective, versatile, and safe targeting and transport of agents. The system is useful in research applications, as well as in the context of translational science and clinical interventions.

Targeted carriers for drug delivery across the gastrointestinal epithelium

A system for targeted delivery of agents (e.g., molecular probes, diagnostic agents, therapeutic agents, imaging agents, research or analytical compounds, enzymes, peptides, proteins, lipids, lipoproteins, sugars, hormones, vitamins, nucleic acids, viruses, bacteria, and/or cells) including use of a composition containing the agent and a targeting moiety, specific for a determinant at the target location. An exemplary composition of the system includes a targeting moiety of one of peptides &#x3b3;3, 2&#x3b3;3, 3&#x3b3;3, A1, B7, B8, B9, B1O, and D6, specific for targeting ICAM-I. The system enables effective, versatile, and safe targeting and transport of agents. The system is useful in research applications, as well as in the context of translational science and clinical interventions.

Peptides for transport of therapeutics and their carriers in mouse models and humans

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Silver Spring, MD, United States of America

Silvia Muro Galindo