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Critical Reviews™ in Eukaryotic Gene Expression

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ISSN Print: 1045-4403

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The Impact Factor measures the average number of citations received in a particular year by papers published in the journal during the two preceding years. 2017 Journal Citation Reports (Clarivate Analytics, 2018) IF: 1.6 To calculate the five year Impact Factor, citations are counted in 2017 to the previous five years and divided by the source items published in the previous five years. 2017 Journal Citation Reports (Clarivate Analytics, 2018) 5-Year IF: 2.2 The Immediacy Index is the average number of times an article is cited in the year it is published. The journal Immediacy Index indicates how quickly articles in a journal are cited. Immediacy Index: 0.3 The Eigenfactor score, developed by Jevin West and Carl Bergstrom at the University of Washington, is a rating of the total importance of a scientific journal. Journals are rated according to the number of incoming citations, with citations from highly ranked journals weighted to make a larger contribution to the eigenfactor than those from poorly ranked journals. Eigenfactor: 0.00058 The Journal Citation Indicator (JCI) is a single measurement of the field-normalized citation impact of journals in the Web of Science Core Collection across disciplines. The key words here are that the metric is normalized and cross-disciplinary. JCI: 0.33 SJR: 0.345 SNIP: 0.46 CiteScore™:: 2.5 H-Index: 67

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Cell Therapy in Duchenne Muscular Dystrophy Treatment: Clinical Trials Overview

Volume 25, Issue 1, 2015, pp. 1-11
DOI: 10.1615/CritRevEukaryotGeneExpr.2015011074
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Anna Bajek
Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland
Dorota Porowinska
Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland
Tomasz Kloskowski
Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland
Edyta Brzoska
Department of Cytology, Institute of Zoology, Faculty of Biology, University of Warsaw, Warsaw, Poland
Maria A. Ciemerych
Department of Cytology, Institute of Zoology, Faculty of Biology, University of Warsaw, Warsaw, Poland
Tomasz Drewa
Chair of Regenerative Medicine, Nicolaus Copernicus University, Bydgoszcz, Poland; Department of Urology, Nicolaus Copernicus Hospital, Torun, Poland

ABSTRACT

Duchenne muscular dystrophy (DMD), the most common and most severe form of all muscular dystrophies, leads to progressive muscle fiber necrosis, fibroblast proliferation, and growth of fibrous tissue and fat. The most common cause of death in DMD patients is cardiac and respiratory failure. Current pharmacological and other treatment methods do not lead to full recovery. For this reason, new alternatives for skeletal muscle regeneration are being investigated. Transplantation of myoblasts from healthy donors is one studied approach to muscle treatment in DMD patients. However, the results of intramuscular injection of in vitro cultured myoblasts are still not satisfactory. The use of autologous stem cells is also proposed. Despite many ongoing studies, this therapy is still in preliminary testing and requires more experiments.

KEY WORDS: Duchenne muscular dystrophy, DMD, cell therapies, myoblast transplantation, muscle regeneration, stem cells
CITED BY

  1. Kowalski Kamil, Kołodziejczyk Aleksandra, Sikorska Maria, Płaczkiewicz Jagoda, Cichosz Paulina, Kowalewska Magdalena, Stremińska Władysława, Jańczyk-Ilach Katarzyna, Koblowska Marta, Fogtman Anna, Iwanicka-Nowicka Roksana, Ciemerych Maria A., Brzoska Edyta, Stem cells migration during skeletal muscle regeneration - the role of Sdf-1/Cxcr4 and Sdf-1/Cxcr7 axis, Cell Adhesion & Migration, 11, 4, 2017. Crossref

  2. Smith Lucas R., Barton Elisabeth R., Regulation of fibrosis in muscular dystrophy, Matrix Biology, 68-69, 2018. Crossref

  3. Wang Yanlin, Hao Lei, Wang Hongcai, Santostefano Katherine, Thapa Arjun, Cleary John, Li Hui, Guo Xiuming, Terada Naohiro, Ashizawa Tetsuo, Xia Guangbin, Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9, Molecular Therapy, 26, 11, 2018. Crossref

  4. Xia Guangbin, Terada Naohiro, Ashizawa Tetsuo, Human iPSC Models to Study Orphan Diseases: Muscular Dystrophies, Current Stem Cell Reports, 4, 4, 2018. Crossref

  5. Mitani Yasuyuki, Vagnozzi Ronald J., Millay Douglas P., In vivo myomaker‐mediated heterologous fusion and nuclear reprogramming , The FASEB Journal, 31, 1, 2017. Crossref

  6. Maurizi Niccolò, Ammirati Enrico, Coppini Raffaele, Morrone Amelia, Olivotto Iacopo, Clinical and Molecular Aspects of Cardiomyopathies, Heart Failure Clinics, 14, 2, 2018. Crossref

  7. Sardone Valentina, Ellis Matthew, Torelli Silvia, Feng Lucy, Chambers Darren, Eastwood Deborah, Sewry Caroline, Phadke Rahul, Morgan Jennifer E., Muntoni Francesco, Asakura Atsushi, A novel high-throughput immunofluorescence analysis method for quantifying dystrophin intensity in entire transverse sections of Duchenne muscular dystrophy muscle biopsy samples, PLOS ONE, 13, 3, 2018. Crossref

  8. Sato Mitsuto, Shiba Naoko, Miyazaki Daigo, Shiba Yuji, Echigoya Yusuke, Yokota Toshifumi, Takizawa Hotake, Aoki Yoshitsugu, Takeda Shin’ichi, Nakamura Akinori, Amelioration of intracellular Ca2+ regulation by exon-45 skipping in Duchenne muscular dystrophy-induced pluripotent stem cell-derived cardiomyocytes, Biochemical and Biophysical Research Communications, 520, 1, 2019. Crossref

  9. Świerczek‐Lasek Barbara, Keremidarska‐Markova Milena, Hristova‐Panusheva Kamelia, Vladkova Todorka, Ciemerych Maria Anna, Archacka Karolina, Krasteva Natalia, Polydimethylsiloxane materials with supraphysiological elasticity enable differentiation of myogenic cells, Journal of Biomedical Materials Research Part A, 107, 12, 2019. Crossref

  10. Tey Sin-Ruow, Robertson Samantha, Lynch Eileen, Suzuki Masatoshi, Coding Cell Identity of Human Skeletal Muscle Progenitor Cells Using Cell Surface Markers: Current Status and Remaining Challenges for Characterization and Isolation, Frontiers in Cell and Developmental Biology, 7, 2019. Crossref

  11. Egorova Tatiana V., Zotova Evgenia D., Reshetov Denis A., Polikarpova Anna V., Vassilieva Svetlana G., Vlodavets Dmitry V., Gavrilov Alexey A., Ulianov Sergey V., Buchman Vladimir L., Deykin Alexei V., CRISPR/Cas9-generated mouse model of Duchenne muscular dystrophy recapitulating a newly identified large 430 kb deletion in the human DMD gene, Disease Models & Mechanisms, 12, 4, 2019. Crossref

  12. Brzoska Edyta, Kalkowski Lukasz, Kowalski Kamil, Michalski Pawel, Kowalczyk Pawel, Mierzejewski Bartosz, Walczak Piotr, Ciemerych Maria A., Janowski Miroslaw, Muscular Contribution to Adolescent Idiopathic Scoliosis from the Perspective of Stem Cell-Based Regenerative Medicine, Stem Cells and Development, 28, 16, 2019. Crossref

  13. Qazi Taimoor H., Duda Georg N., Ort Melanie J., Perka Carsten, Geissler Sven, Winkler Tobias, Cell therapy to improve regeneration of skeletal muscle injuries, Journal of Cachexia, Sarcopenia and Muscle, 10, 3, 2019. Crossref

  14. He Ruojie, Li Huan, Wang Liang, Li Yaqin, Zhang Yu, Chen Menglong, Zhu Yuling, Zhang Cheng, Engraftment of human induced pluripotent stem cell-derived myogenic progenitors restores dystrophin in mice with duchenne muscular dystrophy, Biological Research, 53, 1, 2020. Crossref

  15. Gois Beghini Daniela, Iwao Horita Samuel, Cascabulho Cynthia Machado, Anastácio Alves Luiz, Henriques-Pons Andrea, Induced Pluripotent Stem Cells: Hope in the Treatment of Diseases, including Muscular Dystrophies, International Journal of Molecular Sciences, 21, 15, 2020. Crossref

  16. Świerczek Barbara, Ciemerych Maria A., Archacka Karolina, From pluripotency to myogenesis: a multistep process in the dish, Journal of Muscle Research and Cell Motility, 36, 6, 2015. Crossref

  17. Choi In Young, Lim Ho Tae, Che Young Hyun, Lee Gabsang, Kim Yong Jun, Inhibition of the Combinatorial Signaling of Transforming Growth Factor-Beta and NOTCH Promotes Myotube Formation of Human Pluripotent Stem Cell-Derived Skeletal Muscle Progenitor Cells, Cells, 10, 7, 2021. Crossref

  18. Nakamura Akinori, Moving towards successful exon-skipping therapy for Duchenne muscular dystrophy, Journal of Human Genetics, 62, 10, 2017. Crossref

  19. Duelen Robin, Costamagna Domiziana, Gilbert Guillaume, De Waele Liesbeth, Goemans Nathalie, Desloovere Kaat, Verfaillie Catherine M., Sipido Karin R., Buyse Gunnar M., Sampaolesi Maurilio, Human iPSC model reveals a central role for NOX4 and oxidative stress in Duchenne cardiomyopathy, Stem Cell Reports, 17, 2, 2022. Crossref

  20. Ohlendieck Kay, Swandulla Dieter, Complexity of skeletal muscle degeneration: multi-systems pathophysiology and organ crosstalk in dystrophinopathy, Pflügers Archiv - European Journal of Physiology, 473, 12, 2021. Crossref

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