Hair Follicle: A Novel Source of Stem Cells for Cell and Gene Therapy

2013 ◽  
pp. 97-118
Author(s):  
Panagiotis Mistriotis ◽  
Stelios T. Andreadis
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Hair Follicle ◽  
Cell And Gene Therapy

Cell and gene therapy using mesenchymal stem cells (MSCs)

2008 ◽  
Vol 30 (3) ◽  
pp. 121-127 ◽  
Author(s):  
Keiya Ozawa ◽  
Kazuya Sato ◽  
Iekuni Oh ◽  
Katsutoshi Ozaki ◽  
Ryosuke Uchibori ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Mesenchymal Stem Cells ◽  
Cell And Gene Therapy

Research on cell sources for brain cell replacement methods has gained major importance. Cell and gene therapy are potentially intriguing new domains of regenerative medicine

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Regenerative Medicine ◽  
Brain Cell ◽  
Brain Diseases ◽  
Host Immune System ◽  
Differentiation Potential ◽  
Cell Replacement ◽  
Cell Sources ◽  
Cell And Gene Therapy

Great advances in neurodegenerative disease, cell and gene therapy have been made in recent decades. Following the recent advancement of stem cell-based neuronal therapies, including managing their differentiation potential, research on cell sources for brain cell replacement methods has gained major importance. The objective is to obtain a certain neuronal cell fate to repair and restore the injured cell function. Several cell-based therapeutic techniques that show promise in animal HD models have failed to attain a similar degree of success in human patients. Despite its poor prospects, fetal transplantation has opened the door to a potentially intriguing new domain of regenerative medicine. However, many obstacles need to be overcome before pre-differentiated stem cells can be used in clinical trials, and, in particular, ensuring that the source of stem cells has optimal differentiation potential with full integration and functional enhancement, has measurable clinical benefits with minimal impact on the host immune system, and is tumor-free. New cell, molecular, and pharmacological approaches may assist enhance neuronal survival of transplanted cells, and consequently therapy for many fatal brain diseases. Molecular approaches, on the other hand, have looked into the idea of entirely eliminating HTT utilizing RNAi in the hopes of preventing the mutant protein that produced it in the first place. In contrast, HTT's physiological significance requires the application of procedures that specifically interfere with MHTHTT. The CRISPR/Cas9 approach gives researchers the ability to inactivate the mHTT allele by deleting or editing particular regions, leading to increased knowledge of how to prevent mutation-induced toxicity. Overall, despite their appealing ability to reverse mHTT-induced toxicity, these therapies may face difficulties due to the need to modify their design for individuals in order to ensure therapeutic safety.As clinical investigations are planned, genome editing already shows promise as a potent treatment to overcome clinical HD features. While there is no certainty that HD symptomatology can be fully eased, researchers must continue to hunt for ways to diminish it because it has such profound and life-threatening effects on patients and their families. These new treatments are supposed to bring a brighter future for HD sufferers.

scholarly journals Mesenchymal stem cells at the intersection of cell and gene therapy

2010 ◽  
Vol 10 (12) ◽  
pp. 1663-1679 ◽  
Author(s):  
Timothy J Myers ◽  
Froilan Granero-Molto ◽  
Lara Longobardi ◽  
Tieshi Li ◽  
Yun Yan ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Mesenchymal Stem Cells ◽  
Cell And Gene Therapy

scholarly journals Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing

2021 ◽  
Vol 22 (12) ◽  
pp. 6275
Author(s):  
Dito Anurogo ◽  
Nova Yuli Prasetyo Budi ◽  
Mai-Huong Thi Ngo ◽  
Yen-Hua Huang ◽  
Jeanne Adiwinata Pawitan
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Hematopoietic Stem Cells ◽  
Fanconi Anemia ◽  
Ex Vivo ◽  
Risk Mitigation ◽  
Future Research ◽  
Hematopoietic Stem ◽  
Cell And Gene Therapy

Hereditary anemia has various manifestations, such as sickle cell disease (SCD), Fanconi anemia, glucose-6-phosphate dehydrogenase deficiency (G6PDD), and thalassemia. The available management strategies for these disorders are still unsatisfactory and do not eliminate the main causes. As genetic aberrations are the main causes of all forms of hereditary anemia, the optimal approach involves repairing the defective gene, possibly through the transplantation of normal hematopoietic stem cells (HSCs) from a normal matching donor or through gene therapy approaches (either in vivo or ex vivo) to correct the patient’s HSCs. To clearly illustrate the importance of cell and gene therapy in hereditary anemia, this paper provides a review of the genetic aberration, epidemiology, clinical features, current management, and cell and gene therapy endeavors related to SCD, thalassemia, Fanconi anemia, and G6PDD. Moreover, we expound the future research direction of HSC derivation from induced pluripotent stem cells (iPSCs), strategies to edit HSCs, gene therapy risk mitigation, and their clinical perspectives. In conclusion, gene-corrected hematopoietic stem cell transplantation has promising outcomes for SCD, Fanconi anemia, and thalassemia, and it may overcome the limitation of the source of allogenic bone marrow transplantation.

scholarly journals Therapeutic Effects of Genetically Modified Wharton’s Jelly Mesenchymal Stem Cells Expressing Erythropoietin on Breast Cancer-related Anemia in Mice Model

2021 ◽  
Author(s):  
Mohammad Estiri ◽  
Bahareh Estiri ◽  
Asghar Fallah ◽  
Marzyeh Aghazadeh ◽  
Amir Sedaqati ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Stem Cells ◽  
Gene Therapy ◽  
Mesenchymal Stem Cells ◽  
Lentiviral Vector ◽  
Wharton’S Jelly ◽  
Mice Model ◽  
Wharton's Jelly ◽  
Cell And Gene Therapy ◽  
Cancer Related Anemia

Abstract Cancer-related anemia (CRA) negatively influences cancer patients’ survival, disease progression, treatment efficacy, and quality of life (QOL). Current treatments such as iron therapy, red cell transfusion, and erythropoietin-stimulating agents (ESAs) may cause severe adverse effects including hemolytic transfusion reaction and the possibility of host immunity against rhEPO. Therefore, development of long-lasting and curative therapies is highly required. Combined cell and gene therapy platform can introduce a new route for permanent production of erythropoietin (EPO) in the body with various degrees of clinical benefits and avoiding the need for repeat treatments. In this study, we developed cell and gene therapy strategy for in-vivo delivery of EPO cDNA via genetic engineering human Wharton’s jelly mesenchymal stem cells (hWJMSCs) to long-term produce and secret human EPO protein after transplantation into the mice model of CRA. To evaluate CRA's treatment in cancer-free and cancerous conditions, at first, we designed recombinant breast cancer cell line 4T1 expressing herpes simplex virus type 1 thymidine kinase (HSV1-TK) by a lentiviral vector encoding HSV1-TK and injected into mice. After 3 weeks, all mice develop metastatic breast cancer associated with acute anemia. Then, we administrated ganciclovir (GCV) for 10 days in half of the mice to clear cancer cells. Meanwhile, we designed another lentiviral vector encoding EPO to transduce hWJMSCs. Following implantation of rhWJMSCs-EPO, the whole peripheral blood samples were collected from the tail vein once per week for 10 weeks which were immediately analyzed for the measurements of EPO, hemoglobin (Hb), and hematocrit (Hct) plasma levels. The blood analysis showed that plasma EPO, hemoglobin (Hb), and hematocrit (Hct) concentration significantly increased and remained at a therapeutic level for >10 weeks in both treatment groups which indicates that the rhWJMSCs-EPO could improve CRA in both cancer-free and cancerous mice model.

scholarly journals Therapy Development by Genome Editing of Hematopoietic Stem Cells

Cells ◽  
2021 ◽  
Vol 10 (6) ◽  
pp. 1492
Author(s):  
Lola Koniali ◽  
Carsten W. Lederer ◽  
Marina Kleanthous
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Hematopoietic Stem Cells ◽  
Genome Editing ◽  
Inherited Disorders ◽  
Hematopoietic Stem ◽  
Therapy Targets ◽  
Therapy Development ◽  
Cell And Gene Therapy ◽  
Preclinical And Clinical Studies

Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the blood and immune systems has placed them at the forefront of cell and gene therapy development. Recent advances in genome-editing tools, in particular for clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) and CRISPR/Cas-derived editing systems, have transformed the gene therapy landscape. Their versatility and the ability to edit genomic sequences and facilitate gene disruption, correction or insertion, have broadened the spectrum of potential gene therapy targets and accelerated the development of potential curative therapies for many rare diseases treatable by transplantation or modification of HSCs. Ongoing developments seek to address efficiency and precision of HSC modification, tolerability of treatment and the distribution and affordability of corresponding therapies. Here, we give an overview of recent progress in the field of HSC genome editing as treatment for inherited disorders and summarize the most significant findings from corresponding preclinical and clinical studies. With emphasis on HSC-based therapies, we also discuss technical hurdles that need to be overcome en route to clinical translation of genome editing and indicate advances that may facilitate routine application beyond the most common disorders.

scholarly journals Therapeutic Effects of Genetically Modified Wharton’s Jelly Mesenchymal Stem Cells Expressing Erythropoietin on Cancer-related Anemia in Mice Model

2020 ◽  
Author(s):  
Mohammad Estiri ◽  
Bahareh Estiri ◽  
Asghar Fallah ◽  
Marzyeh Aghazadeh ◽  
Amir Sedaqati ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Stem Cells ◽  
Gene Therapy ◽  
Mesenchymal Stem Cells ◽  
Cancer Cells ◽  
Lentiviral Vector ◽  
Mice Model ◽  
Clinical Benefits ◽  
Cell And Gene Therapy ◽  
Cancer Related Anemia

Abstract BackgroundCancer-related anemia (CRA) negatively influences cancer patients’ survival, disease progression, treatment efficacy, and quality of life (QOL). Current treatments such as iron therapy, red cell transfusion, and erythropoietin-stimulating agents (ESAs) may cause severe adverse effects. Therefore, development of long-lasting and curative therapies is highly required. Combined cell and gene therapy platform can introduce a new route for permanent production of erythropoietin (EPO) in the body with various degrees of clinical benefits and avoiding the need for repeat treatments.MethodsIn this study, we developed cell and gene therapy strategy for in-vivo delivery of EPO cDNA via genetic engineering human Wharton’s jelly mesenchymal stem cells (hWJMSCs) to long-term produce and secret human EPO protein after transplantation into the mice model of CRA. To evaluate CRA's treatment in cancer-free and cancerous conditions, at first, we designed recombinant breast cancer cell line 4T1 expressing herpes simplex virus type 1 thymidine kinase (HSV1-TK) by a lentiviral vector encoding HSV1-TK and injected into mice. After 3 weeks, all mice develop the metastatic breast cancer associated to the acute anemia. Then, we administrated ganciclovir (GCV) for 10 days in half of the mice to clear cancer cells. Meanwhile, we designed another lentiviral vector encoding EPO to transduce hWJMSCs. Following implantation of rhWJMSCs-EPO, the whole peripheral blood samples were collected from the tail vein once per week which were immediately analyzed for the measurements of EPO, hemoglobin (Hb), and hematocrit (Hct) plasma levels. Results We found that after implantation of rhWJMSCs-EPO, plasma EPO, Hb, and Hct concentration significantly increased which rose to a peak in the fourth week and remained at a therapeutic level for >17 weeks in the cancer-free group and >10 weeks in the cancerous group.ConclusionOur data indicate that the EPO-transduced hWJMSCs could improve the anemia of cancer in both cancer-free and cancerous mice model. This significant difference in length of time that Hb and Hct are in therapeutic levels in both treatment groups indicates that developing a precise targeted-therapy to eliminate cancer cells along with an effective treatment for CRA, as we presented here, could bring important clinical benefits.

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