Lizoszomális tárolási rendellenességek kezelési lehetoségei

Translated title of the contribution: Treatment prospects of lysosomal storage disorders

Péter Reismann, Zsolt Tulassay

Research output: Contribution to journalReview article

2 Citations (Scopus)

Abstract

Lysosomal storage disorders are caused either by deficiencies or decreased activity of enzymes localised in lysosomal vesicles or transport failure of these enzymes or their substrates. Accumulation of macromolecules destroy cell function presenting in clinical symptoms. Up to date, there are about 40 different lysosomal storage disorders according to the accumulated macromolecules. Till the last decades supportive therapy was the only option by these disorders. Enhanced researches in the last decades have presented some breakthrough results in the field of storage disease therapy. The review briefly introduces the lysosomal storage disorders, summarizes the actual therapy possibilities, as enzyme replacement therapy, substrate deprivation therapy, bone marrow transplantation. Finally, the review outlines future therapic potentials, like stem-cell and gene therapy.

Translated title of the contributionTreatment prospects of lysosomal storage disorders
Original languageHungarian
Pages (from-to)1171-1179
Number of pages9
JournalOrvosi hetilap
Volume149
Issue number25
DOIs
Publication statusPublished - Jun 22 2008

ASJC Scopus subject areas

  • Medicine(all)

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