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Advances in regenerative therapy: A review of the literature and future directions.

ABSTRACT: There is enormous global anticipation for stem cell-based therapies that are safe and effective. Numerous pre-clinical studies present encouraging results on the therapeutic potential of different cell types including tissue derived stem cells. Emerging evidences in different fields of research suggest several cell types are safe, whereas their therapeutic application and effectiveness remain challenged. Multiple factors that influence treatment outcomes are proposed including immunocompatibility and potency, owing to variations in tissue origin, ex-vivo methodologies for preparation and handling of the cells. This communication gives an overview of literature data on the different types of cells that are potentially promising for regenerative therapy. As a case in point, the recent trends in research and development of the mesenchymal stem cells (MSCs) for cell therapy are considered in detail. MSCs can be isolated from a variety of tissues and organs in the human body including bone marrow, adipose, synovium, and perinatal tissues. However, MSC products from the different tissue sources exhibit unique or varied levels of regenerative abilities. The review finally focuses on adipose tissue-derived MSCs (ASCs), with the unique properties such as easier accessibility and abundance, excellent proliferation and differentiation capacities, low immunogenicity, immunomodulatory and many other trophic properties. The suitability and application of the ASCs, and strategies to improve the innate regenerative capacities of stem cells in general are highlighted among others.

SUBMITTER: Ntege EH

PROVIDER: S-EPMC7033303 | biostudies-literature | 2020 Jun

REPOSITORIES: biostudies-literature

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Advances in regenerative therapy: A review of the literature and future directions.

Ntege Edward H EH Sunami Hiroshi H Shimizu Yusuke Y

Regenerative therapy 20200220

There is enormous global anticipation for stem cell-based therapies that are safe and effective. Numerous pre-clinical studies present encouraging results on the therapeutic potential of different cell types including tissue derived stem cells. Emerging evidences in different fields of research suggest several cell types are safe, whereas their therapeutic application and effectiveness remain challenged. Multiple factors that influence treatment outcomes are proposed including immunocompatibilit ...[more]

PMID: 32110683

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Project description:BackgroundLeber congenital amaurosis (LCA) is a congenital retinal dystrophy that results in significant and often severe vision loss at an early age. Comprehensive analysis of the genetic mutations and phenotypic correlations in LCA patients has allowed for significant improvements in understanding molecular pathways of photoreceptor degeneration and dysfunction. The purpose of this article is to review the literature on the subject of retinal gene therapy for LCA, including historical descriptions, preclinical animal studies, and human clinical trials.MethodsA literature search of peer-reviewed and indexed publications from 1996-2011 using the PubMed search engine was performed. Key terms included "Leber congenital amaurosis", LCA, RPE65, "cone-rod dystrophy", "gene therapy", and "human trials" in various combinations. Seminal articles prior to 1996 were selected from primary sources and reviews from the initial search. Articles were chosen based on pertinence to clinical, genetic, and therapeutic topics reviewed in this manuscript. Fundus photographs from LCA patients were obtained retrospectively from the clinical practice of one of the authors (R.A.S).ResultsHerein, we reviewed the literature on LCA as a genetic disease, the results of human gene therapy trials to date, and possible future directions towards treating inherited retinal diseases at the genetic level. Original descriptions of LCA by Theodor Leber and subsequent research demonstrate the severity of this disease with early-onset blindness. Discoveries of the causative heritable mutations revealed genes and protein products involved in photoreceptor development and visual transduction. Animal models have provided a means to test novel therapeutic strategies, namely gene therapy. Stemming from these experiments, three independent clinical trials tested the safety of subretinal delivery of viral gene therapy to patients with mutations in the RPE65 gene. More recently, efficacy studies have been conducted with encouraging results.ConclusionsInitial safety studies indicated promising results of subretinal delivery of viral vector with subclinical immunologic or surgical sequelae. Overall, these initial studies demonstrate that viral vector gene therapy results are very promising, safe, and effective. Future studies measuring potential improvement in photoreceptor function may rely on recent advances in retinal imaging and electrophysiologic testing.

Project description:BACKGROUND:A new generation of user-centric information systems is emerging in health care as patient health record (PHR) systems. These systems create a platform supporting the new vision of health services that empowers patients and enables patient-provider communication, with the goal of improving health outcomes and reducing costs. This evolution has generated new sets of data and capabilities, providing opportunities and challenges at the user, system, and industry levels. OBJECTIVE:The objective of our study was to assess PHR data types and functionalities through a review of the literature to inform the health care informatics community, and to provide recommendations for PHR design, research, and practice. METHODS:We conducted a review of the literature to assess PHR data types and functionalities. We searched PubMed, Embase, and MEDLINE databases from 1966 to 2015 for studies of PHRs, resulting in 1822 articles, from which we selected a total of 106 articles for a detailed review of PHR data content. RESULTS:We present several key findings related to the scope and functionalities in PHR systems. We also present a functional taxonomy and chronological analysis of PHR data types and functionalities, to improve understanding and provide insights for future directions. Functional taxonomy analysis of the extracted data revealed the presence of new PHR data sources such as tracking devices and data types such as time-series data. Chronological data analysis showed an evolution of PHR system functionalities over time, from simple data access to data modification and, more recently, automated assessment, prediction, and recommendation. CONCLUSIONS:Efforts are needed to improve (1) PHR data quality through patient-centered user interface design and standardized patient-generated data guidelines, (2) data integrity through consolidation of various types and sources, (3) PHR functionality through application of new data analytics methods, and (4) metrics to evaluate clinical outcomes associated with automated PHR system use, and costs associated with PHR data storage and analytics.

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Advances in regenerative therapy: A review of the literature and future directions.

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Advances in regenerative therapy: A review of the literature and future directions.

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