Antibody-Mediated Enzyme Therapeutics and Applications in Glycogen Storage Diseases

Zhengqiu Zhou; Grant L. Austin; Robert Shaffer; Dustin D. Armstrong; Matthew S. Gentry

doi:10.1016/j.molmed.2019.08.005

Antibody-Mediated Enzyme Therapeutics and Applications in Glycogen Storage Diseases

Zhengqiu Zhou
, Grant L. Austin
, Robert Shaffer
, Dustin D. Armstrong
, Matthew S. Gentry

Producción científica: Review article › revisión exhaustiva

35 Citas (Scopus)

Resumen

The use of antibodies as targeting molecules or cell-penetrating tools has emerged at the forefront of pharmaceutical research. Antibody-directed therapies in the form of antibody–drug conjugates, immune modulators, and antibody-directed enzyme prodrugs have been most extensively utilized as hematological, rheumatological, and oncological therapies, but recent developments are identifying additional applications of antibody-mediated delivery systems. A novel application of this technology is for the treatment of glycogen storage disorders (GSDs) via an antibody–enzyme fusion (AEF) platform to penetrate cells and deliver an enzyme to the cytoplasm, nucleus, and/or other organelles. Exciting developments are currently underway for AEFs in the treatment of the GSDs Pompe disease and Lafora disease (LD). Antibody-based therapies are quickly becoming an integral part of modern disease therapeutics.

Idioma original	English
Páginas (desde-hasta)	1094-1109
Número de páginas	16
Publicación	Trends in Molecular Medicine
Volumen	25
N.º	12
DOI	https://doi.org/10.1016/j.molmed.2019.08.005
Estado	Published - dic 2019

Nota bibliográfica

Publisher Copyright:
© 2019 Elsevier Ltd

Financiación

We thank Dr Nadine Aziz of Valerion Therapeutics for acquiring and allowing us to use the immunofluorescence image in Figure I B of Box 2 and for assisting with editing. We thank members of the laboratory of M.S.G. for their support and assistance in critically reviewing this manuscript. M.S.G is funded in part through a sponsored project from Valerion Therapeutics. Additional funding to M.S.G is provided by National Institutes of Health (NIH) grants R01 NS070899 and P01 NS097197, and an Epilepsy Foundation New Therapy Commercialization Grant.

Financiadores	Número del financiador
Epilepsy Foundation New Therapy Commercialization
National Institutes of Health (NIH)	P01 NS097197
National Institutes of Health (NIH)
Institute of Neurological Disorders and Stroke National Advisory Neurological Disorders and Stroke Council	R01NS070899
Institute of Neurological Disorders and Stroke National Advisory Neurological Disorders and Stroke Council
Valerion Therapeutics

ASJC Scopus subject areas

Molecular Medicine
Molecular Biology

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10.1016/j.molmed.2019.08.005

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abstract = "The use of antibodies as targeting molecules or cell-penetrating tools has emerged at the forefront of pharmaceutical research. Antibody-directed therapies in the form of antibody–drug conjugates, immune modulators, and antibody-directed enzyme prodrugs have been most extensively utilized as hematological, rheumatological, and oncological therapies, but recent developments are identifying additional applications of antibody-mediated delivery systems. A novel application of this technology is for the treatment of glycogen storage disorders (GSDs) via an antibody–enzyme fusion (AEF) platform to penetrate cells and deliver an enzyme to the cytoplasm, nucleus, and/or other organelles. Exciting developments are currently underway for AEFs in the treatment of the GSDs Pompe disease and Lafora disease (LD). Antibody-based therapies are quickly becoming an integral part of modern disease therapeutics.",

keywords = "Pompe disease, antibody-directed therapies, Lafora disease, antibody–drug conjugate, antibody–enzyme fusion, glycogen",

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AU - Gentry, Matthew S.

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Antibody-Mediated Enzyme Therapeutics and Applications in Glycogen Storage Diseases

Resumen

Nota bibliográfica

Financiación

ASJC Scopus subject areas

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