ABSTRACT
This article aimed to review current literature on the safety and efficacy of stem cell therapy in Stargardt disease. A comprehensive literature search was performed, and two animal and eleven human clinical trials were retrieved. These studies utilized different kinds of stem cells, including human or mouse embryonic stem cells, mesenchymal stem cells, bone marrow mononuclear fraction, and autologous bone marrow-derived stem cells. In addition, different injection techniques including subretinal, intravitreal, and suprachoroidal space injections have been evaluated. Although stem cell therapy holds promise in improving visual function in patients with Stargardt disease, further investigation is needed to determine the long-term benefits, safety, and efficacy in determining the best delivery method and selecting the most appropriate stem cell type.
Subject(s)
Stargardt Disease , Stem Cells , Review Literature as Topic , Vitelliform Macular Dystrophy , Macular DegenerationABSTRACT
L'autogreffe de cellules souches hématopoïétiques (CSH) est une technique thérapeutique, permettant d'utiliser les cellules souches du patient afin de contourner l'écueil de l'aplasie sévère post chimiothérapie. Son coût de revient très élevé l'a rendue inaccessible dans de nombreux pays et en particulier en Afrique où on n'en compte que six pays avec 16 centres par rapport à 679 en Europe, ce qui représente un déficit de 98%. En Algérie, la première greffe de cellules a été effectuée en 1998 au niveau du CPMC d'Alger. Le 2ème centre de greffe de CSH a vu le jour en 2009 à l'EHU 1er Novembre d'Oran et est devenu un centre de greffe à vocation nationale. Nous présentons dans ce travail, le cheminement de la mise en place de la procédure de greffe de cellules à Oran, selon les recommandations internationales et leurs adaptations selon les conditions locales de travail.
Subject(s)
Stem Cells , Transplantation, Autologous , Hematopoietic Stem Cells , TransplantationABSTRACT
Neurological disease encompasses a diverse group of disorders of the central and peripheral nervous systems, which collectively are the leading cause of disease burden globally. The scope of treatment options for neurological disease is limited, and drug approval rates for improved treatments remain poor when compared with other therapeutic areas. Stem cell therapy provides hope for many patients, but should be tempered with the realisation that the scientific and medical communities are still to fully unravel the complexities of stem cell biology, and to provide satisfactory data that support the rational, evidence-based application of these cells from a therapeutic perspective. We provide an overview of the application of stem cells in neurological disease, starting with basic principles, and extending these to describe the clinical trial landscape and progress made over the last decade. Many forms of stem cell therapy exist, including the use of neural, haematopoietic and mesenchymal stem cells. Cell therapies derived from differentiated embryonic stem cells and induced pluripotent stem cells are also starting to feature prominently. Over 200 clinical studies applying various stem cell approaches to treat neurological disease have been registered to date (Clinicaltrials.gov), the majority of which are for multiple sclerosis, stroke and spinal cord injuries. In total, we identified 17 neurological indications in clinical stage development. Few studies have progressed into large, pivotal investigations with randomised clinical trial designs. Results from such studies will be essential for approval and application as mainstream treatments in the future
Subject(s)
Cell- and Tissue-Based Therapy , Nervous System Diseases , South Africa , Stem CellsABSTRACT
Stem cells are basic cells of all multicellular organisms having the potency to differentiate into wide range of adult cells. Self renewal and totipotency are characteristic of stem cells. Though totipotency is shown by very early embryonic stem cells; the adult stem cells possess multipotency and differential plasticity which can be exploited for future generation of therapeutic options. Fortunately; the regulators of pleuripotency such as oct-4 et nanong protein are discovered and possibility of in vitro regulation of pleuripotency of stem cells is gaining strength. Genetic regulation of adult stem cells in the form of Bmi-1; Notch; sonic hedgehog et wnt gene is also being worked upon and future can be regulation of stem cell differentiation in vitro; in vivo or both. It is the knowledge of regulators of stem cells which has opened the therapeutic usage of stem cells in the form of neuron regeneration; treatment of bone defect; drug testing; gene therapy and cell based therapy in the form of muscle damage; spinal cord injury; cancer therapy etc. Cell based therapies might become commercial in coming years