Exploiting the diphtheria toxin internalization receptor enhances delivery of proteins to lysosomes for enzyme replacement therapy-Reference-Cited by-同舟云学术

Exploiting the diphtheria toxin internalization receptor enhances delivery of proteins to lysosomes for enzyme replacement therapy

Published:2020-12-11 Issue:50 Volume:6 Page:
ISSN:2375-2548
Container-title:Science Advances
language:en
Short-container-title:Sci. Adv.

Author:

Sugiman-Marangos Seiji N.¹^ORCID,Beilhartz Greg L.¹^ORCID,Zhao Xiaochu²^ORCID,Zhou Dongxia³,Hua Rong³⁴,Kim Peter K.³⁴^ORCID,Rini James M.³⁵^ORCID,Minassian Berge A.²⁶^ORCID,Melnyk Roman A.¹³^ORCID

Affiliation:

1. Molecular Medicine Program, The Hospital for Sick Children, Toronto, ON, Canada.

2. Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada.

3. Department of Biochemistry, University of Toronto, Toronto, ON, Canada.

4. Cell Biology Program, The Hospital for Sick Children, 686 Bay Street, Toronto, ON, Canada.

5. Department of Molecular Genetics, University of Toronto, ON M5S1A8, Canada.

6. Department of Pediatrics and Dallas Children’s Medical Center, University of Texas Southwestern, Dallas, TX 75390-9063, USA.

Abstract

Engineering lysosomal enzymes to bind the diphtheria toxin receptor with high affinity increases uptake into cells and tissues.

Funder

Canadian Institutes of Health Research

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

Reference39 articles.

1. Lysosomal storage disorders: The cellular impact of lysosomal dysfunction

2. Lysosomal Storage Diseases: From Pathophysiology to Therapy

3. Enzyme Replacement Therapy for Lysosomal Diseases: Lessons from 20 Years of Experience and Remaining Challenges

4. The Phosphomannosyl Recognition System for Intracellular and Intercellular Transport of Lysosomal Enzymes

5. Ligand interactions of the cation-dependent mannose 6-phosphate receptor

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