scholarly journals The Use of Stem Cells to Treat Osteogenesis Imperfecta

2019 ◽  
Vol 3 (2) ◽  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Osteogenesis Imperfecta ◽  
Bone Structure ◽  
Genetic Disorder ◽  
Treatment Options ◽  
Collagen Matrix ◽  
Current Treatment ◽  
Distant Future ◽  
Strength And Durability

Osteogenesis Imperfecta is a genetic disorder that affects the body’s ability to create and maintain bone decreasing its strength and durability due to mutations in specific genes that code for the creation of the collagen matrix in the bone structure. Current treatment options available for individuals suffering from Osteogenesis Imperfecta include conventional rehabilitation therapy as well as drug therapy. Recently research has focused on finding a cure using gene therapy, thus having the ability to correct the mutations that cause the weakened collagen fibers. In addition, research is focusing on the use of stem cells that have the ability to differentiate into osteoblasts that can create new and stronger bone. The transplantation of mesenchymal stem cells has been tested to treat the disorder. Positive results have been reported when transplanted prenatally and/or postnatally, through the current use of stem cells and gene therapy using murine models. Additionally, a few trials performed in humans are being planned, in the not too distant future. Once a sufficient number of trials are completed with reported success, treatments using stem cells should be available to patients with Osteogenesis Imperfecta.

scholarly journals Current Overview on the Use of Mesenchymal Stem Cells for Perianal Fistula Treatment in Patients with Crohn’s Disease

Life ◽  
2021 ◽  
Vol 11 (11) ◽  
pp. 1133
Author(s):  
Marcin Włodarczyk ◽  
Katarzyna Czerwińska ◽  
Jakub Włodarczyk ◽  
Jakub Fichna ◽  
Adam Dziki ◽  
...  
Keyword(s):  
Stem Cells ◽  
Mesenchymal Stem Cells ◽  
Crohn’S Disease ◽  
Crohn's Disease ◽  
Treatment Options ◽  
Perianal Fistula ◽  
Current Treatment ◽  
Culture Conditions ◽  
Optimal Dosage ◽  
Number Of Patients

Perianal fistula in patients with Crohn’s disease is an extremely challenging condition. The disease tends to reoccur, and with current treatment options, a large number of patients are left with active ailment and experience major morbidity. In recent years, hopeful results regarding local use of mesenchymal stem cells (MSCs) in perianal Crohn’s disease have been published. Although to this day there are no clear guidelines determining optimal dosage, injections frequency and culture conditions, their efficiency has proven to be much higher than conventionally used methods. According to studies, they can effectively induce as well as maintain fistula closure. This approach also avoids common side effects related to conventional surgical treatment.

scholarly journals Filling the Gap: Neural Stem Cells as A Promising Therapy for Spinal Cord Injury

2019 ◽  
Vol 12 (2) ◽  
pp. 65 ◽  
Author(s):  
Inês M. Pereira ◽  
Ana Marote ◽  
António J. Salgado ◽  
Nuno A. Silva
Keyword(s):  
Spinal Cord ◽  
Stem Cells ◽  
Spinal Cord Injury ◽  
Clinical Trials ◽  
Neural Stem Cells ◽  
Treatment Options ◽  
Experimental Studies ◽  
Current Treatment ◽  
Surgical Interventions ◽  
Cord Injury

Spinal cord injury (SCI) can lead to severe motor, sensory and social impairments having a huge impact on patients’ lives. The complex and time-dependent SCI pathophysiology has been hampering the development of novel and effective therapies. Current treatment options include surgical interventions, to stabilize and decompress the spinal cord, and rehabilitative care, without providing a cure for these patients. Novel therapies have been developed targeting different stages during trauma. Among them, cell-based therapies hold great potential for tissue regeneration after injury. Neural stem cells (NSCs), which are multipotent cells with inherent differentiation capabilities committed to the neuronal lineage, are especially relevant to promote and reestablish the damaged neuronal spinal tracts. Several studies demonstrate the regenerative effects of NSCs in SCI after transplantation by providing neurotrophic support and restoring synaptic connectivity. Therefore, human clinical trials have already been launched to assess safety in SCI patients. Here, we review NSC-based experimental studies in a SCI context and how are they currently being translated into human clinical trials.

scholarly journals Distal renal tubular acidosis: a systematic approach from diagnosis to treatment

2021 ◽  
Author(s):  
Sabrina Giglio ◽  
Giovanni Montini ◽  
Francesco Trepiccione ◽  
Giovanni Gambaro ◽  
Francesco Emma
Keyword(s):  
Metabolic Acidosis ◽  
Renal Tubular Acidosis ◽  
Molecular Mechanisms ◽  
Clinical Symptoms ◽  
Genetic Disorder ◽  
Treatment Options ◽  
Current Treatment ◽  
Hydrogen Ions ◽  
Renal Tubular

AbstractRenal tubular acidosis (RTA) comprises a group of disorders in which excretion of hydrogen ions or reabsorption of filtered HCO3is impaired, leading to chronic metabolic acidosis with normal anion gap. In the current review, the focus is placed on the most common type of RTA, Type 1 RTA or Distal RTA (dRTA), which is a rare chronic genetic disorder characterized by an inability of the distal nephron to secrete hydrogen ions in the presence of metabolic acidosis. Over the years, knowledge of the molecular mechanisms behind acid secretion has improved, thereby greatly helping the diagnosis of dRTA. The primary or inherited form of dRTA is mostly diagnosed in infancy, childhood, or young adulthood, while the acquired secondary form, as a consequence of other disorders or medications, can happen at any age, although it is more commonly seen in adults. dRTA is not as “benign” as previously assumed, and can have several, highly variable long-term consequences. The present review indeed reports and summarizes both clinical symptoms and diagnosis, long-term outcomes, genetic inheritance, epidemiology and current treatment options, with the aim of shedding more light onto this rare disorder. Being a chronic condition, dRTA also deserves attention in the transition between pediatric and adult nephrology care, and as a rare disease it has a place in the European and Italian rare nephrological diseases network.

Asthma immunotherapy and treatment approaches with mesenchymal stem cells

Immunotherapy ◽  
2020 ◽  
Vol 12 (9) ◽  
pp. 665-674
Author(s):  
Tunç Akkoç ◽  
Deniz Genç
Keyword(s):  
Stem Cells ◽  
Mesenchymal Stem Cells ◽  
Specific Immunotherapy ◽  
Treatment Options ◽  
Treatment Strategies ◽  
Chronic Inflammatory Disease ◽  
Current Treatment ◽  
Dependent Manner ◽  
Allergen Specific Immunotherapy ◽  
And Control

Asthma is a chronic inflammatory disease of the airways where exaggerated T helper 2 immune responses and inflammatory mediators play a role. Current asthma treatment options can effectively suppress symptoms and control the inflammatory process; however, cannot modulate the dysregulated immune response. Allergen-specific immunotherapy is one of the effective treatments capable of disease modification. Injecting allergens under the skin in allergen-specific immunotherapy can reduce asthma and improve the sensitivity of the lungs, however, has a risk of severe reactions. Mesenchymal stem cells have immunoregulatory activity with their soluble mediators and contact dependent manner. In this review, we focus on the current treatment strategies with mesenchymal stem cells in asthma as a new therapeutic tool and compare those with immunotherapy.

Feasibility of Targeting Glioblastoma Stem Cells: From Concept to Clinical Trials

2020 ◽  
Vol 19 (32) ◽  
pp. 2974-2984 ◽  
Author(s):  
Vadim V. Tarasov ◽  
Andrey A. Svistunov ◽  
Vladimir N. Chubarev ◽  
Tamara A. Zatsepilova ◽  
Nina G. Preferanskaya ◽  
...  
Keyword(s):  
Stem Cells ◽  
Clinical Trials ◽  
Cancer Stem Cells ◽  
Treatment Options ◽  
Current Treatment ◽  
Clinical Settings ◽  
Glioblastoma Stem Cells ◽  
Pubmed Search ◽  
Number Of Treatment ◽  
Target Cancer

Objective: Glioblastoma is a highly aggressive and invasive brain and Central Nervous System (CNS) tumor. Current treatment options do not prolong overall survival significantly because the disease is highly prone to relapse. Therefore, research to find new therapies is of paramount importance. It has been discovered that glioblastomas contain a population of cells with stem-like properties and that these cells are may be responsible for tumor recurrence. Methods: A review of relevant papers and clinical trials in the field was conducted. A PubMed search with related keywords was used to gather the data. For example, “glioblastoma stem cells AND WNT signaling” is an example used to find information on clinical trials using the database ClinicalTrials.gov. Results: Cancer stem cell research has several fundamental issues and uncertainties that should be taken into consideration. Theoretically, a number of treatment options that target glioblastoma stem cells are available for patients. However, only a few of them have obtained promising results in clinical trials. Several strategies are still under investigation. Conclusion: The majority of treatments to target cancer stem cells have failed during clinical trials. Taking into account a number of biases in the field and the number of unsuccessful investigations, the application of the cancer stem cells concept is questionable in clinical settings, at least with respect to glioblastoma.

scholarly journals Overnight transduction with foamyviral vectors restores the long-term repopulating activity of Fancc−/− stem cells

Blood ◽  
2008 ◽  
Vol 112 (12) ◽  
pp. 4458-4465 ◽  
Author(s):  
Yue Si ◽  
Anna C. Pulliam ◽  
Yvonne Linka ◽  
Samantha Ciccone ◽  
Cordula Leurs ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Bone Marrow ◽  
Stem Cell ◽  
Ex Vivo ◽  
Bone Marrow Failure ◽  
Genetic Disorder ◽  
Cell Phenotype ◽  
Functional Correction ◽  
Complex Genetic Disorder

AbstractFanconi anemia (FA) is a complex genetic disorder characterized by congenital abnormalities, bone marrow failure, and myeloid malignancies. Identification of 13 FA genes has been instrumental to explore gene transfer technologies aimed at correction of autologous FA-deficient stem cells. To date, 3 human FA stem cell gene therapy trials with standard 4-day transduction protocols using gammaretroviral vectors failed to provide clinical benefit. In addition, 2- to 4 day ex vivo manipulation of bone marrow from mice containing a disruption of the homologue of human FANCC (Fancc) results in a time-dependent increase in apoptosis and a risk for malignant transformation of hematopoietic cells. Here, we show that a 14-hour transduction period allows a foamyviral vector construct expressing the human FANCC cDNA to efficiently transduce murine FA stem cells with 1 to 2 proviral integrations per genome. Functionally, the repopulating activity of Fancc−/− stem cells from reconstituted mice expressing the recombinant FANCC transgene was comparable with wild-type controls. Collectively, these data provide evidence that short-term transduction of c-kit+ cells with a foamyviral vector is sufficient for functional correction of a stem cell phenotype in a murine FA model. These data could have implications for future gene therapy trials for FA patients.

scholarly journals New Approaches to Treat Osteoarthritis with Mesenchymal Stem Cells

2018 ◽  
Vol 2018 ◽  
pp. 1-9 ◽  
Author(s):  
Kiran Shah ◽  
Ashley G. Zhao ◽  
Huseyin Sumer
Keyword(s):  
Stem Cells ◽  
Mesenchymal Stem Cells ◽  
Weight Bearing ◽  
Treatment Options ◽  
Current Treatment ◽  
Cartilage Volume ◽  
Pharmacological Intervention ◽  
Translational Medical Research ◽  
Reduced Mobility ◽  
Chronic Health Problems

Osteoarthritis is one of the most common chronic health problems in the world that causes disability and chronic pain with reduced mobility and is a progressive degenerative disease in weight-bearing joints such as the knee. The pathology of the joint resulting from OA includes loss of cartilage volume and cartilage lesions leading to inflammation of the articular joint structures; its incidence and progression are associated with a variety of risk factors. Most of the current treatments focus on symptom management such as physical and occupational therapies, pharmacological intervention for pain management, and surgical intervention with limited success and do not address nor halt the progression of the disease. In this review, we will describe the current treatment options for OA and the exciting new translational medical research currently underway utilising mesenchymal stem cells for OA therapy.

Treatment of Vocal Fold Scarring: Rheological and Histological Measures of Homologous Collagen Matrix

2002 ◽  
Vol 111 (10) ◽  
pp. 884-889 ◽  
Author(s):  
Susan L. Thibeault ◽  
Patrick J. VanGroll ◽  
Kevin J. Kriesel ◽  
Roger W. Chan ◽  
Diane M. Bless ◽  
...  
Keyword(s):  
Lamina Propria ◽  
Vocal Fold ◽  
Dynamic Properties ◽  
Treatment Options ◽  
Collagen Matrix ◽  
Vocal Folds ◽  
Biomechanical Properties ◽  
Treated Group ◽  
Current Treatment ◽  
Muscular Tissue

The current treatment options for dysphonia secondary to vocal fold scarring are limited. Few studies address changes in the lamina propria, which is critical to vocal fold biomechanical properties and voice production. Using rheological and histological measures of homologous collagen matrix (HCM)–injected vocal folds, we assessed HCM's potential for providing bulk and restoring biomechanical performance. Twenty rabbits underwent bilateral vocal fold scarring. After 10 weeks of scar maturation, the rabbits had unilateral injections of HCM or saline solution. Ten weeks after the injections, histological studies revealed well-defined collagen globules distributed throughout the lamina propria and underlying muscular tissue. Significantly more procollagen was observed in the HCM-treated group. No significant differences in elastic shear modulus or dynamic viscosity were found between the treatment groups. This study demonstrates that HCM is an inert, relatively stable injectate that may serve well for medialization but does not appear to improve the dynamic properties of the lamina propria.

An Overview of Dementias

2012 ◽  
Vol 21 (3) ◽  
pp. 75-84
Author(s):  
Venkata Vijaya K. Dalai ◽  
Jason E. Childress ◽  
Paul E Schulz
Keyword(s):  
Clinical Presentation ◽  
Treatment Options ◽  
Current Treatment ◽  
Great Variability ◽  
Health Concern ◽  
Public Health Concern ◽  
Life Threatening ◽  
The Common ◽  
Neurodegenerative Dementias ◽  
Made In

Dementia is a major public health concern that afflicts an estimated 24.3 million people worldwide. Great strides are being made in order to better diagnose, prevent, and treat these disorders. Dementia is associated with multiple complications, some of which can be life-threatening, such as dysphagia. There is great variability between dementias in terms of when dysphagia and other swallowing disorders occur. In order to prepare the reader for the other articles in this publication discussing swallowing issues in depth, the authors of this article will provide a brief overview of the prevalence, risk factors, pathogenesis, clinical presentation, diagnosis, current treatment options, and implications for eating for the common forms of neurodegenerative dementias.

1417: Mesenchymal Stem Cells Alone or Modified with ENOS Restore Erectile Function in the Aged Rat: Cell-Based Gene Therapy for Erectile Dysfunction

2004 ◽  
Vol 171 (4S) ◽  
pp. 373-373
Author(s):  
Trinity J. Bivalacqua ◽  
Mustafa F. Usta ◽  
Hunter C. Champion ◽  
Weiwen Deng ◽  
Philip J. Kadowitz ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Mesenchymal Stem Cells ◽  
Erectile Dysfunction ◽  
Erectile Function ◽  
Aged Rat
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