Erratum to: Gene Editing, Gene Therapy, and Cell Xenotransplantation: Cell Transplantation Across Species

Source: Current Transplantation Reports - Category: Transplant Surgery Source Type: research

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AbstractHaematopoietic stem cells (HSCs) are the only adult stem cells with a demonstrated clinical use, even though a tractable method to maintain and expand human HSCsin vitro has not yet been found. Owing to the introduction of transplantation strategies for the treatment of haematological malignancies and, more recently, the promise of gene therapy, the need to improve the generation, manipulation, and scalability of autologous or allogeneic HSCs has risen steeply over the past decade. In that context, reprogramming strategies based on the expression of exogenous transcription factors have emerged as a means to produce...
Source: FEBS Letters - Category: Biochemistry Authors: Tags: Review Source Type: research
Abstract Over the last decade, incrementally improved xenograft mouse models, which support the engraftment and development of a human hemato-lymphoid system, have been developed and represent an important fundamental and preclinical research tool. Immunodeficientmicecan be transplanted with human hematopoietic stem cells (HSCs) and this process is accompanied by HSC homing to the murine bone marrow. This is followed by stem cell expansion, multilineage hematopoiesis, long-term engraftment, and functional human antibody and cellular immune responses. The most significant contributions made by these humanized mice ...
Source: Biochemical Pharmacology - Category: Drugs & Pharmacology Authors: Tags: Biochem Pharmacol Source Type: research
In conclusion, high-dose NR induces the onset of WAT dysfunction, which may in part explain the deterioration of metabolic health. Towards a Rigorous Definition of Cellular Senescence https://www.fightaging.org/archives/2019/11/towards-a-rigorous-definition-of-cellular-senescence/ The accumulation of lingering senescent cells is a significant cause of aging, disrupting tissue function and generating chronic inflammation throughout the body. Even while the first senolytic drugs capable of selectively destroying these cells already exist, and while a number of biotech companies are working on the productio...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
A few higher animal species, such as salamanders and zebrafish, are capable of regeneration of limbs and internal organs, regrowing lost and injured tissue without scarring or loss of function. Numerous research groups are engaged in investigating the biochemistry of proficient regeneration, attempting to find the specific differences between species that might explain how it happens and why adult mammals are largely incapable of such feats of regrowth. Today's open access research is an example of the type, in which the authors narrow down on a specific cell population that appear in zebrafish hearts during regeneration, ...
Source: Fight Aging! - Category: Research Authors: Tags: Medicine, Biotech, Research Source Type: blogs
Purpose of review We briefly address the advances in genetics, pathophysiology, and phenotypes of chronic granulomatous disease (CGD). This is one of the most studied primary immunodeficiencies, which comprise mutations in genes encoding the different subunits of the NADPH oxidase system. Those mutations lead to defective reactive oxygen species production, and consequently a failure to eliminate pathogens. Recent findings Patients with CGD are susceptible to fungal, bacterial, and parasitic infections. Other symptoms, as systemic adverse effects to BCG vaccine and hyperinflammation, are also important clinical condit...
Source: Current Opinion in Pediatrics - Category: Pediatrics Tags: ALLERGY, IMMUNOLOGY AND RELATED DISORDERS: Edited by Jordan S. Orange Source Type: research
Conclusion. Given the unique genetic mutations reported in PID patients on the African continent and the feasibility of hematopoietic stem cell transplantation and gene therapy, increased awareness should be encouraged and new therapeutic options considered. PMID: 31662142 [PubMed - in process]
Source: South African Medical Journal - Category: African Health Tags: S Afr Med J Source Type: research
Three researchers at the  Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA have received awards totaling more than $18 million from the California Institute for Regenerative Medicine, the state’s stem cell agency. The recipients areDr. Sophie Deng, professor of ophthalmology at the UCLA Stein Eye Institute;  Yvonne Chen, a UCLA associate professor of microbiology, immunology and molecular genetics; andDr. Caroline Kuo, a UCLA assistant clinical professor of pediatrics. The awards were announced at a CIRM meeting today.Deng ’s four-year, $10.3 million award ...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news
In this study, the enhanced mice live somewhat longer than their unmodified peers, though not as much longer as is the case for the application of telomerase gene therapy. The mice do also exhibit reduced cancer risk, however. The scientists here class telomere shortening as a cause of aging, which is not a point universally agreed upon. Reductions in average telomere length in tissues looks much more like a downstream consequence of reduced stem cell activity than an independent mechanism. Researchers obtain the first mice born with hyper-long telomeres and show that it is possible to extend life without any geneti...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Publication date: Available online 25 October 2019Source: Stem Cell ResearchAuthor(s): Lionel O. Mavoungou, Samuel Neuenschwander, Uyen Pham, Pavithra S. Iyer, Nicolas MermodABSTRACTDuchenne muscular dystrophy (DMD) is a lethal muscle-wasting disease caused by the lack of dystrophin in muscle fibers that is currently without curative treatment. Mesoangioblasts (MABs) are multipotent progenitor cells that can differentiate to a myogenic lineage and that can be used to express Dystrophin upon transplantation into muscles, in autologous gene therapy approaches. However, their fate in the muscle environment remains poorly...
Source: Stem Cell Research - Category: Stem Cells Source Type: research
Abstract Intrabodies (both single-chain Fv and single-domain VH, VHH, and VL nanobodies) offer unique solutions to some of the challenges of delivery and target engagement posed by immunotherapeutics for the brain and other areas of the nervous system. The specificity, which includes the recognition of post-translational modifications, and capacity for engineering that characterize these antibody fragments can be especially well-focused when the genes encoding only the binding sites of the antibody are expressed intracellularly. Multifunctional constructs use fusions with peptides that can re-target antigen-antibo...
Source: Neurobiology of Disease - Category: Neurology Authors: Tags: Neurobiol Dis Source Type: research
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