Influence of Immune Responses in Gene/Stem Cell Therapies for Muscular Dystrophies

Farini, Andrea; Sitzia, Clementina; Erratico, Silvia; Meregalli, Mirella; Torrente, Yvan

doi:10.1155/2014/818107

Cited by 10 publications

(11 citation statements)

References 196 publications

(231 reference statements)

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“…In addition, human IgG might also be able to regulate the immune response in gene therapeutic approaches (Farini et al . ) and, thus, provide a suitable addition to the standard corticosteroid treatment.…”

Section: Discussionmentioning

confidence: 99%

Treatment with human immunoglobulin G improves the early disease course in a mouse model of Duchenne muscular dystrophy

Zschüntzsch

Zhang

Klinker

et al. 2015

Journal of Neurochemistry

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Duchenne muscular dystrophy (DMD) is a severe hereditary myopathy. Standard treatment by glucocorticosteroids is limited because of numerous side effects. The aim of this study was to test immunomodulation by human immunoglobulin G (IgG) as treatment in the experimental mouse model (mdx) of DMD. 2 g/ kg human IgG compared to human albumin was injected intraperitoneally in mdx mice at the age of 3 and 7 weeks. Advanced voluntary wheel running parameters were recorded continuously. At the age of 11 weeks, animals were killed so that blood, diaphragm, and lower limb muscles could be removed for quantitative PCR, histological analysis and ex vivo muscle contraction tests. IgG compared to albumin significantly improved the voluntary running performance and reduced muscle fatigability in an ex vivo muscle contraction test. Upon IgG treatment, serum creatine kinase values were diminished and mRNA expression levels of relevant inflammatory markers were reduced in the diaphragm and limb muscles. Macrophage infiltration and myopathic damage were significantly ameliorated in the quadriceps muscle. Collectively, this study demonstrates that, in the early disease course of mdx mice, human IgG improves the running performance and diminishes myopathic damage and inflammation in the muscle. Therefore, IgG may be a promising approach for treatment of DMD.

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Section: Discussionmentioning

confidence: 99%

Treatment with human immunoglobulin G improves the early disease course in a mouse model of Duchenne muscular dystrophy

Zschüntzsch

Zhang

Klinker

et al. 2015

Journal of Neurochemistry

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“…First, they can differentiate into muscular progenitors and myocytes, which can fuse with damaged tissue and restore expression of dystrophin, as was shown in the mdx mouse model of DMD 27 . Second, they secrete immunomodulatory, anti‐inflammatory, antiapoptotic, neovascular, and proangiogenic factors, such as cytokines, chemokines, or growth factors, which improve milieu by decreasing inflammation, increasing oxygen supply, and ameliorating trophic conditions 28,29 . Third, they improve the neural component of the disease.…”

Section: Introductionmentioning

confidence: 94%

The Use of Umbilical Cord-Derived Mesenchymal Stem Cells in Patients with Muscular Dystrophies: Results from Compassionate Use in Real-Life Settings

Świątkowska-Flis

Zdolińska-Malinowska

Sługocka

et al. 2021

Stem Cells Translational Medicine

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Muscular dystrophies are genetically determined progressive diseases with no causerelated treatment and limited supportive treatment. Although stem cells cannot resolve the underlying genetic conditions, their wide-ranging therapeutic properties may ameliorate the consequences of the involved mutations (oxidative stress, inflammation, mitochondrial dysfunction, necrosis). In this study, we administered advanced therapy medicinal product containing umbilical cord-derived mesenchymal stem cells (UC-MSCs)

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“…The advantage of gene therapy is that the continuous expression of genes can increase the endogenous gene products in target tissues, such as signal molecules and transcription factors ( 3 ). Compared with stem cell therapy, gene therapy has a lower incidence of immune responses ( 88 ). Gene therapy can produce continuous local production and secretion of proteins.…”

Section: Treatment Of Tendon Injuriesmentioning

confidence: 99%

Basic Research on Tendon Repair: Strategies, Evaluation, and Development

Yang

Zhou

2021

Front. Med.

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Tendon is a fibro-elastic structure that links muscle and bone. Tendon injury can be divided into two types, chronic and acute. Each type of injury or degeneration can cause substantial pain and the loss of tendon function. The natural healing process of tendon injury is complex. According to the anatomical position of tendon tissue, the clinical results are different. The wound healing process includes three overlapping stages: wound healing, proliferation and tissue remodeling. Besides, the healing tendon also faces a high re-tear rate. Faced with the above difficulties, management of tendon injuries remains a clinical problem and needs to be solved urgently. In recent years, there are many new directions and advances in tendon healing. This review introduces tendon injury and sums up the development of tendon healing in recent years, including gene therapy, stem cell therapy, Platelet-rich plasma (PRP) therapy, growth factor and drug therapy and tissue engineering. Although most of these therapies have not yet developed to mature clinical application stage, with the repeated verification by researchers and continuous optimization of curative effect, that day will not be too far away.

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Influence of Immune Responses in Gene/Stem Cell Therapies for Muscular Dystrophies

Cited by 10 publications

References 196 publications

Treatment with human immunoglobulin G improves the early disease course in a mouse model of Duchenne muscular dystrophy

Treatment with human immunoglobulin G improves the early disease course in a mouse model of Duchenne muscular dystrophy

The Use of Umbilical Cord-Derived Mesenchymal Stem Cells in Patients with Muscular Dystrophies: Results from Compassionate Use in Real-Life Settings

Basic Research on Tendon Repair: Strategies, Evaluation, and Development

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