Drug delivery systems in Krabbe disease—present and prospective approaches

Prabitha Priyadharshini, A; Umamaheswari, A; Vijayalakshmi, M; Chellappan, DK; Dua, K; Prabu, SL

Drug delivery systems in Krabbe disease—present and prospective approaches

Prabitha Priyadharshini, A Umamaheswari, A Vijayalakshmi, M Chellappan, DK Dua, K

Prabu, SL

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Publisher:: Elsevier
Publication Type:: Chapter
Citation:: Drug Delivery Systems for Metabolic Disorders, 2022, pp. 317-336
Issue Date:: 2022-01-01

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Field	Value	Language
dc.contributor.author	Prabitha Priyadharshini, A
dc.contributor.author	Umamaheswari, A
dc.contributor.author	Vijayalakshmi, M
dc.contributor.author	Chellappan, DK
dc.contributor.author	Dua, K https://orcid.org/0000-0002-7507-1159
dc.contributor.author	Prabu, SL
dc.date.accessioned	2023-02-24T22:21:00Z
dc.date.available	2023-02-24T22:21:00Z
dc.date.issued	2022-01-01
dc.identifier.citation	Drug Delivery Systems for Metabolic Disorders, 2022, pp. 317-336
dc.identifier.isbn	9780323996334
dc.identifier.uri	http://hdl.handle.net/10453/166425
dc.description.abstract	Lysosomal enzymes help in the degradation of various complex biomolecules. Mutations arising from genes encoding them result in deficiency of these enzymes causing serious disturbances in specific metabolic pathways. Krabbe Disease (KD), is one such condition, where galactocerebrosidase enzyme deficiency occurs which is critical for galactosylceramide (GalCer) degradation, which if present abundantly results in a toxic secondary metabolite psychosine accumulation in myelin. It also incites globoid cell infiltration, ultimately resulting in myelination cessation affecting both central nervous system (CNS) and peripheral nervous system. Current life-supportive treatments are not curative since accompanied limitations impede attaining better therapeutic efficacy. Also, the complex pathogenic cascade makes addressing the target difficult. Therapeutic efficacy can be improved by designing ideal drug delivery systems with potential therapeutic agents. This chapter discusses current treatment modalities followed in KD and challenges associated along with a few novel therapeutic approaches which might provide leads for treatment improvements and advancements.
dc.language	en
dc.publisher	Elsevier
dc.relation.ispartof	Drug Delivery Systems for Metabolic Disorders
dc.relation.isbasedon	10.1016/B978-0-323-99616-7.00030-X
dc.rights	info:eu-repo/semantics/closedAccess
dc.title	Drug delivery systems in Krabbe disease—present and prospective approaches
dc.type	Chapter
pubs.organisational-group	/University of Technology Sydney
pubs.organisational-group	/University of Technology Sydney/Faculty of Health
pubs.organisational-group	/University of Technology Sydney/Faculty of Health/Graduate School of Health
pubs.organisational-group	/University of Technology Sydney/Faculty of Health/Graduate School of Health/GSH.Pharmacy
utslib.copyright.status	closed_access	*
dc.date.updated	2023-02-24T22:20:59Z
pubs.publication-status	Published

Abstract:

Lysosomal enzymes help in the degradation of various complex biomolecules. Mutations arising from genes encoding them result in deficiency of these enzymes causing serious disturbances in specific metabolic pathways. Krabbe Disease (KD), is one such condition, where galactocerebrosidase enzyme deficiency occurs which is critical for galactosylceramide (GalCer) degradation, which if present abundantly results in a toxic secondary metabolite psychosine accumulation in myelin. It also incites globoid cell infiltration, ultimately resulting in myelination cessation affecting both central nervous system (CNS) and peripheral nervous system. Current life-supportive treatments are not curative since accompanied limitations impede attaining better therapeutic efficacy. Also, the complex pathogenic cascade makes addressing the target difficult. Therapeutic efficacy can be improved by designing ideal drug delivery systems with potential therapeutic agents. This chapter discusses current treatment modalities followed in KD and challenges associated along with a few novel therapeutic approaches which might provide leads for treatment improvements and advancements.

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http://hdl.handle.net/10453/166425