Stem Cell Applications in Lysosomal Storage Disorders: Progress and Ongoing Challenges

dc.authorid köse, sevil/0000-0003-2188-9534
dc.authorid Çetinkaya, Duygu Uçkan/0000-0003-3593-6493
dc.authorid Aerts Kaya, Fatima/0000-0002-9583-8572
dc.authorid KORKUSUZ, PETEK/0000-0002-7553-3915
dc.authorscopusid 56494407000
dc.authorscopusid 36439180500
dc.authorscopusid 7004861894
dc.authorscopusid 6602104436
dc.authorwosid köse, sevil/ABI-5227-2020
dc.authorwosid Çetinkaya, Duygu Uçkan/X-2067-2019
dc.authorwosid Aerts Kaya, Fatima/ABG-1880-2020
dc.contributor.author Kose, S.
dc.contributor.author Aerts-Kaya, F.
dc.contributor.author Cetinkaya, D. Uckan
dc.contributor.author Korkusuz, P.
dc.contributor.other Nutrition and Dietetics
dc.date.accessioned 2024-07-05T15:18:41Z
dc.date.available 2024-07-05T15:18:41Z
dc.date.issued 2021
dc.department Atılım University en_US
dc.department-temp [Kose, S.] Atilim Univ, Dept Med Biol, Fac Med, Ankara, Turkey; [Aerts-Kaya, F.; Cetinkaya, D. Uckan] Hacettepe Univ, Dept Stem Cell Sci, Grad Sch Hlth Sci, Ankara, Turkey; [Aerts-Kaya, F.] Hacettepe Univ, Ctr Stem Cell Res & Dev PEDI STEM, Ankara, Turkey; [Cetinkaya, D. Uckan] Hacettepe Univ, Fac Med, Dept Pediat, Ctr Stem Cell Res & Dev PEDI STEM,Div Hematol, Ankara, Turkey; [Korkusuz, P.] Hacettepe Univ, Fac Med, Dept Histol & Embryol, Ankara, Turkey en_US
dc.description köse, sevil/0000-0003-2188-9534; Çetinkaya, Duygu Uçkan/0000-0003-3593-6493; Aerts Kaya, Fatima/0000-0002-9583-8572; KORKUSUZ, PETEK/0000-0002-7553-3915 en_US
dc.description.abstract Lysosomal storage disorders (LSDs) are rare inborn errors of metabolism caused by defects in lysosomal function. These diseases are characterized by accumulation of completely or partially degraded substrates in the lysosomes leading to cellular dysfunction of the affected cells. Currently, enzyme replacement therapies (ERTs), treatments directed at substrate reduction (SRT), and hematopoietic stem cell (HSC) transplantation are the only treatment options for LSDs, and the effects of these treatments depend strongly on the type of LSD and the time of initiation of treatment. However, some of the LSDs still lack a durable and curative treatment. Therefore, a variety of novel treatments for LSD patients has been developed in the past few years. However, despite significant progress, the efficacy of some of these treatments remains limited because these therapies are often initiated after irreversible organ damage has occurred. Here, we provide an overview of the known effects of LSDs on stem cell function, as well as a synopsis of available stem cell-based cell and gene therapies that have been/are being developed for the treatment of LSDs. We discuss the advantages and disadvantages of use of hematopoietic stem cell (HSC), mesenchymal stem cell (MSC), and induced pluripotent stem cell (iPSC)-related (gene) therapies. An overview of current research data indicates that when stem cell and/or gene therapy applications are used in combination with existing therapies such as ERT, SRT, and chaperone therapies, promising results can be achieved, showing that these treatments may result in alleviation of existing symptoms and/or prevention of progression of the disease. All together, these studies offer some insight in LSD stem cell biology and provide a hopeful perspective for the use of stem cells. Further development and improvement of these stem cell (gene) combination therapies may greatly improve the current treatment options and outcomes of patients with a LSD. en_US
dc.description.sponsorship Turkish Ministry of Development [2006-K120640]; Scientific and Technological Research Council of Turkey (TUBITAK) [219S675] en_US
dc.description.sponsorship This study was supported by a grant from the Turkish Ministry of Development, PediSTEM nr. 2006-K120640, and the Scientific and Technological Research Council of Turkey (TUBITAK), project nr. 219S675. en_US
dc.identifier.citationcount 2
dc.identifier.doi 10.1007/5584_2021_639
dc.identifier.endpage 162 en_US
dc.identifier.isbn 9783030804923
dc.identifier.isbn 9783030804916
dc.identifier.issn 0065-2598
dc.identifier.issn 2214-8019
dc.identifier.pmid 33977438
dc.identifier.scopus 2-s2.0-85122448886
dc.identifier.startpage 135 en_US
dc.identifier.uri https://doi.org/10.1007/5584_2021_639
dc.identifier.uri https://hdl.handle.net/20.500.14411/1891
dc.identifier.volume 1347 en_US
dc.identifier.wos WOS:000796201100008
dc.institutionauthor Köse, Sevil
dc.language.iso en en_US
dc.publisher Springer international Publishing Ag en_US
dc.relation.ispartofseries Advances in Experimental Medicine and Biology
dc.relation.publicationcategory Kitap Bölümü - Uluslararası en_US
dc.rights info:eu-repo/semantics/closedAccess en_US
dc.scopus.citedbyCount 3
dc.subject Lysosomal storage disorder en_US
dc.subject Lysosomal storage disease en_US
dc.subject Stem cell en_US
dc.subject Hematopoietic stem cell en_US
dc.subject Mesenchymal stem cell en_US
dc.subject Neural stem cell en_US
dc.subject Induced pluripotent stem cell en_US
dc.subject Gene therapy en_US
dc.title Stem Cell Applications in Lysosomal Storage Disorders: Progress and Ongoing Challenges en_US
dc.type Book Part en_US
dc.wos.citedbyCount 3
dspace.entity.type Publication
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