Induced Pluripotent Stem Cells: Therapeutic Applications in Monogenic and Metabolic Diseases, and Regulatory and Bioethical Considerations

Liras, Antonio; Segovia, Carlos; Gabán, Aline S.

doi:10.5772/55816

Cited by 6 publications

(3 citation statements)

References 120 publications

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“…Stem cell therapy is evolving as a new medical application concept in pharmacology. For all practical purposes, human embryonic stem cells are used in 13% of treatments, whereas fetal stem cells are used in 2%, umbilical cord stem cells in 10%, and adult stem cells in 75% of cases [48]. We found one study of mouse, based on this study, all authors conclude that cell therapy using genetically engineered endothelial progenitor and stem cells can generate functional coagulation factor VIII (FVIII) and provide stable long-term therapy for hemophilia A.…”

Section: Stem Cell Therapymentioning

confidence: 88%

Hemophilia: Diagnosis and Innovative Management.

Saiyed,

Rangrej

2023

PIJR

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Hemophilia is an acquired,X-linked,passive disorder caused by an insufficiency of utilitarian plasma clotting factor VIII (FVIII). In a critical number of cases, the disorder results from a novo mutation. It rarely manifests as an acquired autoimmune process.Research facility thinks about for suspected hemophilia include a total blood cell tally,coagulation ponders, and an FVIII assay. In patients with an established diagnosis of hemophilia, periodic research facility evaluations include screening for the presence of FVIII inhibitors and screening for transfusion-related or transmissible illnesses such as hepatitis and HIV contamination. Estimation of FVIII levels is critical for observing FVIII substitution treatment. Treatment of hemophilia includes prophylaxis, treatment of bleeding episodes, and induction of immune tolerance.Intended for the treatment and rehabilitation of the patients who are taking factor inhibitors and patients have hemophilic synovitis. Treatment for hemophiliacs should ideally be provided by a Hemophilia Day Care center.We discuss innovative management for the prevention of hemophilia through gene therapy,non-replacement therapy,EHL recombinant concentration,etc.we discuss the data from our studies and emerging results from other gene therapy trials in both hemophilia A and B. This analysis will provide evidence and information on the feasibility and data quality/completeness of the hemophilia database to assess the impact of innovative management on hemophilia outcomes in developing countries.We used a statistical method to analyze the data of adult and pediatric patients who come to hemophilia day care center for taking treatment.The data analysis was performed by the data collected monthly at the hemophilia day care center.

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Section: Stem Cell Therapymentioning

confidence: 88%

Hemophilia: Diagnosis and Innovative Management.

Saiyed,

Rangrej

2023

PIJR

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“…iPSCs technology opens a wide range of therapeutic possibilities, employing different strategies for different necessities. Functional cells could be engrafted, either as stem cells or differentiated to another cell type, to replace the damaged ones [37]. It is also possible that injected stem cells secrete growth factors stimulating the repair of non-functional cells [38].…”

Section: The Potential Of Ips Cell-based Therapeuticsmentioning

confidence: 99%

The Challenge of Bringing iPSCs to the Patient

Ortuño-Costela

Cerrada

García-López

et al. 2019

IJMS

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The implementation of induced pluripotent stem cells (iPSCs) in biomedical research more than a decade ago, resulted in a huge leap forward in the highly promising area of personalized medicine. Nowadays, we are even closer to the patient than ever. To date, there are multiple examples of iPSCs applications in clinical trials and drug screening. However, there are still many obstacles to overcome. In this review, we will focus our attention on the advantages of implementing induced pluripotent stem cells technology into the clinics but also commenting on all the current drawbacks that could hinder this promising path towards the patient.In this review, we summarize the main applications of iPSCs in the clinics, the progress achieved to date, and the path towards the patient, including the possible drawbacks that might appear in this process.

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“…Based on their potency and their sources, stem cells can generally be classified into totipotent, multipotent and pluripotent types (Table 1) [1,2]. Induced pluripotent stem cells (iPSCs) have certain identification properties and specific stringency criteria both in human as well as in mouse model systems (Table 2) [3][4][5].…”

Section: Introductionmentioning

confidence: 99%

Induced Pluripotent Stem Cells and Their Future Therapeutic Applications in Hematology

Al-Anazi¹

2015

J Stem Cell Res Ther

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Reprogramming of iPSCs should have the following requirements: (1) species such as humans or mice, (2) cell type such as fibroblasts or blood cells, (3) factor or chemical such as protein, gene or valproic acid, (4) vector such as retrovirus or lentivirus, and (5) diseases with specific genetic mutations [6,7,9]. DNA and non-DNA methods have been employed in induction and programming of iPSCs (Table 4) [4-9]. The following genes have been used in inducing PSCs: Oct 3/4, Sox2, Klf4, C-Myc, Nanog, LIN 28, Glis 1 and REX 1 [3,4,7]. Also, a long list of agents or vectors have been used in iPSC programming and these include: single or multiple transient transfections, excisable and nonintegrating vectors, proteins and direct protein transduction, plasmid and episomal vectors, minicircle DNA, modified RNA, RNA-based Sendai viruses, mRNA-based transcription factor delivery, microRNA transfections, artificial chromosome vectors, chemical compounds and small molecule compounds. These agents have been reported to improve safety and efficacy of the reprogramming process [5,7]. Small molecule compounds have recently been utilized to generate mouse iPSCs from

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Induced Pluripotent Stem Cells: Therapeutic Applications in Monogenic and Metabolic Diseases, and Regulatory and Bioethical Considerations

Cited by 6 publications

References 120 publications

Hemophilia: Diagnosis and Innovative Management.

Hemophilia: Diagnosis and Innovative Management.

The Challenge of Bringing iPSCs to the Patient

Induced Pluripotent Stem Cells and Their Future Therapeutic Applications in Hematology

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