Adenovirus vectors in hematopoietic stem cell genome editing

AbstractGenome editing of hematopoietic stem cells (HSCs) represents a therapeutic option for a number of hematological genetic diseases, as HSCs have the potential for self ‐renewal potential and differentiation into all blood cell lineages. This review presents advances of genome editing in HSCs utilizing adenovirus vectors as delivery vehicles. We focus on capsid‐modified, helper‐dependent adenovirus vectors that are devoid of all viral genes and therefore exhi bit an improved safety profile. We discuss HSC genome engineering for several inherited disorders and infectious diseases including hemoglobinopathies, Fanconi anemia, hemophilia and HIV‐1 infection byex vivo andin vivo editing in transgenic mice, non ‐human primates, as well as in human CD34+ cells. Mechanisms of therapeutic gene transfer, including episomal expression of designer nucleases and base editors, transposase ‐mediated random integration, and targeted homology‐directed repair triggered integration into selected genomic safe harbour loci are also reviewed.
Source: FEBS Letters - Category: Biochemistry Authors: Tags: REVIEW Source Type: research

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The purpose of this study was to explore the difference in clinical outcome of 2-strand and 4-strand flexor tendon repairs in a single unit in adult population. A total of 109 complete divisions of a single flexor tendon were analyzed from 2016 to 2018 retrospectively. Thirty flexor tendons were repaired with 2-strand and 79 tendons were repaired with 4-strand technique. There was no significant difference in the complication rate including rupture, infection, and adhesions. These results support that 4-strand is not superior than 2-strand and that lower volume type of repair would be preferable.
Source: Annals of Plastic Surgery - Category: Cosmetic Surgery Tags: Hand Surgery Source Type: research
Conclusion Based on photogrammetry, primary cleft nasal correction in our patients with unilateral cleft lip achieved acceptable and stable outcomes during early childhood.
Source: Annals of Plastic Surgery - Category: Cosmetic Surgery Tags: Head and Neck Surgery Source Type: research
DNA repair genes can be used as prognostic biomarkers in many types of cancer. We aimed to identify prognostic DNA repair genes in patients with gastric cancer (GC) by systematically bioinformatic approaches using web-based database. Global gene expression profiles from altogether 1,325 GC patients' samples from six independent datasets were included in the study. Clustering analysis was performed to screen potentially abnormal DNA repair genes related to the prognosis of GC, followed by unsupervised clustering analysis to identify molecular subtypes of GC. Characteristics and prognosis differences were analyzed among thes...
Source: Journal of Cancer - Category: Cancer & Oncology Authors: Tags: Research Paper Source Type: research
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Source: Journal of Cancer - Category: Cancer & Oncology Authors: Tags: Research Paper Source Type: research
Conclusions: Under the induction of tumor microenvironment, MSCs can differentiate into myofibroblasts and then affect tumor interstitial microenvironment remodeling. This process is mediated by TGFβ1-Smad2 pathway.
Source: Journal of Cancer - Category: Cancer & Oncology Authors: Tags: Research Paper Source Type: research
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Source: Journal of Cancer - Category: Cancer & Oncology Authors: Tags: Research Paper Source Type: research
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Source: Journal of Cancer - Category: Cancer & Oncology Authors: Tags: Research Paper Source Type: research
Background: Actinins are major cytoskeletal proteins that mediate sarcomere function, and they also have important non-muscle functions such as regulating cytokinesis, cell adhesion and migration. There are four isoforms of actinins in mammals (ACTN1-4). Recently, the relationship between actinins and cancer has been discovered in many types of malignancy, yet their prognostic significance in acute myeloid leukemia (AML) remains unclear.Methods: We collected data of 155 de novo AML patients from The Cancer Genome Atlas (TCGA) database; 85 patients received chemotherapy only and 70 patients underwent allogeneic hematopoieti...
Source: Journal of Cancer - Category: Cancer & Oncology Authors: Tags: Research Paper Source Type: research
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Source: Journal of Cancer - Category: Cancer & Oncology Authors: Tags: Research Paper Source Type: research
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Source: Journal of Cancer - Category: Cancer & Oncology Authors: Tags: Research Paper Source Type: research
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