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Lysosomal Storage Disorders and Enzyme Replacement Therapy
Published in Peter Grunwald, Pharmaceutical Biocatalysis, 2020
Mikulka and Sands (2016) have summarized the actual treatment approaches for Krabbe disease. Hematopoietic stem cell transplantation serves to delay disease progression, but is not an effective cure. Enzyme replacement therapies will fail because rGalC does not cross the blood-brain barrier. Other investigations dealt with viral-mediated gene-therapy, substrate reduction therapy and several anti-inflammatory therapies, etc. All these single-treatment methods resulted, if at all, in moderate success. Hence, a combination of treatment strategies with the aim to target multiple pathogenic mechanisms/pathways in conjunction with HSCT turned out more successful in pre-clinical testing. Rather recently, Marshall et al. (2018) published results of a global adeno-associated virus (AAV)9-based gene therapy protocol to deliver therapeutic GalC to newborn mice affected by GALC deficiency (twitcher mice). This treatment led to nearly complete correction of GalC’s metabolic deficiencies across the entire nervous system and showed long-term protection of demyelination, neuroinflammation, and motor function.
Treatment of Rheumatoid Arthritis
Published in Richard K. Burt, Alberto M. Marmont, Stem Cell Therapy for Autoimmune Disease, 2019
Stuart Weisman, Arthur Kavanaugh
During the past decade, the approach to the treatment of patients with rheumatoid arthritis (RA) has undergone remarkable change. Non steroidal anti-inflammatory drugs (NSAIDs), corticosteroids, and conventional disease modifying anti-rheumatic drugs (DMARDs), including methotrexate, sulfasalazine, hydroxychloroquine, and others continue to be used successfully in some patients. However, the introduction of highly effective treatments, including leflunomide and the biological response modifiers, etanercept, infliximab, and anakinra, have dramatically altered therapy for patients with rheumatoid arthritis. Some recent studies evaluating combination of therapies have also shown substantial efficacy. These advances will be discussed in this chapter. Hematopoietic stem cell transplantation will be discussed in Chapter 43.
Cellular Biology in Tissue Engineering
Published in Joseph W. Freeman, Debabrata Banerjee, Building Tissues, 2018
Joseph W. Freeman, Debabrata Banerjee
Essentially, prevention and treatment are two main goals of immunosuppressive therapy. Drugs are designed to inhibit T cell activation and effector functions. However, one important issue with drugs is that they provide non-specific immunosuppression, and thus patients become more susceptible to infections (especially to intracellular microbes) and also have increased incidence of cancer. This is because of the depletion of circulating lymphocytes in the body. More specifically, when dealing with cancers such as leukemia, hematopoietic stem cell transplantation can be performed. The first step is to ablate the existing bone marrow to create room for the recipient cells. This causes a deficiency in erythrocytes and B-cell lymphocytes. This makes the person temporarily vulnerable because of their severely compromised immune system. It is critical to monitor the patient for the time being until the transplant is accepted.
The effects of mineral trioxide aggregate on osteo/odontogenic potential of mesenchymal stem cells: a comprehensive and systematic literature review
Published in Biomaterial Investigations in Dentistry, 2020
Danial Babaki, Sanam Yaghoubi, Maryam M. Matin
While cell and molecular signalings involved in the migration of dental pulp progenitor cells during natural reparative dentinogenesis are not fully understood, recent studies focused on strategies mimicking these natural phenomena [53–57]. Similar to well-known hematopoietic stem cell transplantation procedure, it has faced many difficulties in developing regenerative approaches. These challenges include choosing the best cell source, means of differentiation and lineage potency, and the plasticity of the stem cells [58,59]. Well documented results of pulp capping with MTA suggested that it can provide a surface for adhesion of progenitor cells. Moreover, its low-level cytotoxicity, paracrine effects, and potential to induce expression of osteo/odontoblast gene markers in adult tissue-derived stem cells make MTA an applicable chemical for stem cell-based therapies in dentin-pulp regeneration [60–62].