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Advances in stem cell therapy for amyotrophic lateral sclerosis
Maggiore Carita Univ Hosp Novara, Dept Neurol, ALS Ctr, Novara, Italy.
Univ Milano Bicocca, Dept Biotechnol & Biosci, Milan, Italy.
Univ Belgrade, Fac Biol, Ctr Laser Microscopy, Belgrade, Serbia.
Polish Acad Sci, Mossakowski Med Res Ctr, Stem Cell Bioengn Unit, Warsaw, Poland.
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2018 (English)In: Expert Opinion on Biological Therapy, ISSN 1471-2598, E-ISSN 1744-7682, Vol. 18, no 8, p. 865-881Article, review/survey (Refereed) Published
Abstract [en]

Introduction: Amyotrophic Lateral Sclerosis (ALS) is a progressive, incurable neurodegenerative disease that targets motoneurons. Cell-based therapies have generated widespread interest as a potential therapeutic approach but no conclusive results have yet been reported either from pre-clinical or clinical studies. Areas covered: This is an integrated review of pre-clinical and clinical studies focused on the development of cell-based therapies for ALS. We analyze the biology of stem cell treatments and results obtained from pre-clinical models of ALS and examine the methods and the results obtained to date from clinical trials. We discuss scientific, clinical, and ethical issues and propose some directions for future studies. Expert opinion: While data from individual studies are encouraging, stem-cell-based therapies do not yet represent a satisfactory, reliable clinical option. The field will critically benefit from the introduction of well-designed, randomized and reproducible, powered clinical trials. Comparative studies addressing key issues such as the nature, properties, and number of donor cells, the delivery mode and the selection of proper patient populations that may benefit the most from cell-based therapies are now of the essence. Multidisciplinary networks of experts should be established to empower effective translation of research into the clinic.

Place, publisher, year, edition, pages
2018. Vol. 18, no 8, p. 865-881
Keywords [en]
Amyotrophic lateral sclerosis, stem cells, transplantation, clinical trials, animal models, cellular models
National Category
Cell and Molecular Biology Medical Biotechnology (with a focus on Cell Biology (including Stem Cell Biology), Molecular Biology, Microbiology, Biochemistry or Biopharmacy)
Identifiers
URN: urn:nbn:se:uu:diva-366394DOI: 10.1080/14712598.2018.1503248ISI: 000445196000004PubMedID: 30025485OAI: oai:DiVA.org:uu-366394DiVA, id: diva2:1265126
Available from: 2018-11-22 Created: 2018-11-22 Last updated: 2018-11-22Bibliographically approved

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Kozlova, Elena

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