keyword
https://read.qxmd.com/read/38534877/effect-of-fibrillin-2-on-differentiation-into-periodontal-ligament-stem-cell-like-cells-derived-from-human-induced-pluripotent-stem-cells
#1
JOURNAL ARTICLE
Sayuri Hamano, Daiki Yamashita, Daigaku Hasegawa, Hideki Sugii, Tomohiro Itoyama, Hidefumi Maeda
Periodontal tissue regeneration is important for preserving teeth. Periodontal ligament stem cells (PDLSCs) are useful in periodontal tissue regeneration, however tooth extraction is required to obtain these cells. Therefore, we focused on induced pluripotent stem (iPS) cells and established a method to obtain PDLSC-like cells from iPS cells. Specifically, we first differentiated iPS cells into neural crest-like cells (iNCs). Next, we obtained PDLSC-like cells (iPDLSCs) by culturing iNCs on extracellular matrix (ECM) derived from human primary periodontal ligament cells (HPDLCs)...
March 27, 2024: Stem Cells and Development
https://read.qxmd.com/read/38517255/effect-of-allogeneic-hematopoietic-stem-cell-transplantation-on-sickle-cell-disease-related-organ-complications-a-systematic-review-and-meta-analysis
#2
REVIEW
Elisabeth Dovern, Mesire Aydin, Michael R DeBaun, Komeil Alizade, Bart J Biemond, Erfan Nur
Sickle cell disease (SCD)-related organ complications are a major cause of morbidity and mortality in patients with SCD. We sought to assess whether hematopoietic stem cell transplantation (HSCT) stabilizes, attenuates, or exacerbates organ decline. We performed a systematic review and meta-analysis of trials investigating organ function before and after HSCT in patients with SCD. We searched MEDLINE/PubMed and EMBASE up to September 21, 2023. Continuous data were expressed as standardized mean difference (SMD) and pooled in a weighted inverse-variance random-effects model; binomial data were expressed as risk ratio (RR) using the Mantel-Haenszel random-effects meta-analyses...
March 22, 2024: American Journal of Hematology
https://read.qxmd.com/read/38516178/a-scoping-review-exploring-cure-definitions-and-language-for-inherited-hemoglobinopathies
#3
JOURNAL ARTICLE
Marilyn S Baffoe-Bonnie, K Jameson Floyd, Alicia A Livinski, Christine Grady
PURPOSE: Sickle cell disease and beta thalassemia are some of the first targets for potentially curative cell-based therapies. Currently, bone marrow transplants, stem cell transplants, and gene therapy are being researched and utilized for people living with these hemoglobinopathies. Although these therapies are often described as curative, there is not a clear definition of what cure means for these hemoglobinopathies. METHODS: Five databases were searched for this scoping review...
2024: Genet Med Open
https://read.qxmd.com/read/38506770/ex-vivo-culture-resting-time-impacts-transplantation-outcomes-of-genome-edited-human-hematopoietic-stem-and-progenitor-cells-in-xenograft-mouse-models
#4
JOURNAL ARTICLE
Selami Demirci, Muhammad B N Khan, Gabriela Hinojosa, Anh Le, Alexis Leonard, Khaled Essawi, Bjorg Gudmundsdottir, Xiong Liu, Jing Zeng, Zaina Inam, Rebecca Chu, Naoya Uchida, Daisuke Araki, Evan London, Henna Butt, Stacy A Maitland, Daniel E Bauer, Scot A Wolfe, Andre Larochelle, John F Tisdale
Ex vivo resting culture is a standard procedure following genome editing in hematopoietic stem and progenitor cells (HSPCs). However, prolonged culture may critically affect cell viability and stem cell function. We investigated whether varying durations of culture resting times impact the engraftment efficiency of human CD34+ HSPCs edited at the BCL11A enhancer, a key regulator in the expression of fetal hemoglobin. We employed electroporation to introduce CRISPR-Cas9 components for BCL11A enhancer editing and compared outcomes with nonelectroporated (NEP) and electroporated-only (EP) control groups...
February 24, 2024: Cytotherapy
https://read.qxmd.com/read/38493007/revolutionising-healing-gene-editing-s-breakthrough-against-sickle-cell-disease
#5
REVIEW
Marija Dimitrievska, Dravie Bansal, Marta Vitale, John Strouboulis, Annarita Miccio, Kypros H Nicolaides, Sara El Hoss, Panicos Shangaris, Joanna Jacków-Malinowska
Recent advancements in gene editing illuminate new potential therapeutic approaches for Sickle Cell Disease (SCD), a debilitating monogenic disorder caused by a point mutation in the β-globin gene. Despite the availability of several FDA-approved medications for symptomatic relief, allogeneic hematopoietic stem cell transplantation (HSCT) remains the sole curative option, underscoring a persistent need for novel treatments. This review delves into the growing field of gene editing, particularly the extensive research focused on curing haemoglobinopathies like SCD...
March 7, 2024: Blood Reviews
https://read.qxmd.com/read/38488686/favorable-outcome-of-non-myeloablative-allogeneic-transplantation-in-adult-patients-with-severe-sickle-cell-disease-a-single-center-experience-of-200-patients
#6
JOURNAL ARTICLE
Moussab Damlaj, Bader Alahmari, Ahmed Alaskar, Ayman Alhejazi, Husam Alsadi, Mazin Ahmed, Tahani Alanazi, Rasha Ahmed, Amani Alharbi, Inaam Shehabeddine, Afnan Alzaidi, Suha Alkhuraisat, Isam Mahassnah, Hamza Alquraan, Maybelle Ballili, Mohsen Alzahrani
Allogeneic hematopoietic stem cell transplant (HSCT) for adults with severe sickle cell disease (SCD) is potentially curative but not commonly utilized therapy due to complications such as graft failure (GF) and organ toxicity. Herein, we are reporting our long-term outcome data of non-myeloablative (NMA) HSCT in adults with severe SCD with emphasis on factors predicting event free survival (EFS). Adults with severe SCD undergoing NMA match-related donor allogeneic HSCT from 2015 to 2021 with at least 12 months of follow-up were included...
March 15, 2024: American Journal of Hematology
https://read.qxmd.com/read/38486114/durable-engraftment-after-pharmacological-pre-transplant-immune-suppression-followed-by-reduced-toxicity-myeloablative-haploidentical-stem-cell-transplantation-in-highly-hla-immunized-adults-with-sickle-cell-disease
#7
JOURNAL ARTICLE
Sabine Fürst, Emmanuelle Bernit, Faezeh Legrand, Angela Granata, Samia Harbi, Raynier Devillier, Valerio Maisano, Benjamin Bouchacourt, Thomas Pagliardini, Djamel Mokart, Claude Lemarié, Boris Calmels, Christophe Picard, Agnès Basire, Borje S Andersson, Didier Blaise
Allogeneic stem cell transplantation (Allo-SCT) is the only rapidly available curative treatment modality in patients with severe sickle cell disease (SCD). The development of reduced-toxicity myeloablative conditioning (RT-MAC) regimen and the use of partially matched family donors with post-transplantation cyclophosphamide (PT-Cy) have widened the access to Allo-SCT. Antibodies against donor-specific HLA (DSA) increase the risk of engraftment failure in HLA mismatched Allo-SCT. We report the results of five patients with SCD, whereas three with DSA, who underwent an unmanipulated haploidentical stem cell transplantation (Haplo-SCT) after a busulfan-based RT-MAC regimen with PT-Cy...
March 14, 2024: Bone Marrow Transplantation
https://read.qxmd.com/read/38445379/the-essence-of-quiescence
#8
JOURNAL ARTICLE
Peter J Quesenberry, Mark S Dooner, Mandy Pereira, Nathalie Oulhen, Sicheng Wen
Historically hematopoietic stem cells are believed to be predominantly dormant but could be induced into active cell cycle under specific conditions. This review, coupled with years of research from our laboratory, challenges this belief by demonstrating a significant portion of hematopoietic stem cells are actively cycling rather than quiescent. This addresses a major heuristic error in the understanding of hematopoietic stem cells which has shaped this field for decades. By evaluating the cycle status of engraftable hematopoietic stem cells in whole unseparated bone marrow, we demonstrated that a significant portion of these cells are actively cycling, and further confirmed by tritiated thymidine suicide and BrdU labeling assays...
March 6, 2024: Stem Cells and Development
https://read.qxmd.com/read/38445374/biodistribution-and-safety-of-human-multi-chimeric-cells-hmcc-after-systemic-intraosseous-and-intravenous-administration-in-the-experimental-mouse-model
#9
JOURNAL ARTICLE
Maria Siemionow, Lucile Chambily, Joanna Cwykiel
Cellular therapies provide promising options for inducing tolerance in transplantation of solid organs, bone marrow, and vascularized composite allografts. However, novel tolerance-inducing protocols remain limited despite extensive research. We previously introduced and characterized a Human Multi-Chimeric Cell (HMCC) line, created through ex vivo fusion of human umbilical cord blood (UCB) cells derived from three unrelated donors. In this study, we assessed in vivo biodistribution and safety of HMCC in the NOD...
March 6, 2024: Stem Cells and Development
https://read.qxmd.com/read/38390839/survival-and-functional-integration-of-human-embryonic-stem-cell-derived-retinal-organoids-after-shipping-and-transplantation-into-retinal-degeneration-rats
#10
JOURNAL ARTICLE
Bin Lin, Ratnesh Singh, Magdalene J Seiler, Igor O Nasonkin
Because derivation of retinal organoids (ROs) and transplantation are frequently split between geographically distant locations, we have developed a special shipping device and protocol capable of the organoids' delivery to any location. Human embryonic stem cell (hESC) derived ROs were differentiated from the hESC line H1 (WA01), shipped overnight to another location, and then transplanted into the subretinal space of blind immunodeficient retinal degenerate (RD) rats. Development of transplants was monitored by spectral-domain optical coherence tomography...
February 23, 2024: Stem Cells and Development
https://read.qxmd.com/read/38386545/extracellular-vesicles-a-new-avenue-of-mesenchymal-stem-cell-therapies-in-transplant-medicine
#11
JOURNAL ARTICLE
Steven Levitte
No abstract text is available yet for this article.
February 20, 2024: Stem Cells and Development
https://read.qxmd.com/read/38386508/effects-of-seipin-on-mouse-mesenchymal-stem-cell-osteo-adipogenic-balance
#12
JOURNAL ARTICLE
Zeying Li, Shan Jin, Tong Xu, Hongzhi Chen, Wenping Cai, Jin Du, Qiu Jin, Sihui Zhuang, Yan Qi, Wenyi Gu, Lijuan Pang
Seipin deficiency is an important cause of type 2 Berardinelli-Seip congenital dyslipidemia (BSCL2). BSCL2 is a severe lipodystrophy syndrome with lack of adipose tissue, hepatic steatosis, insulin resistance, and normal or higher bone mineral density. Bone marrow mesenchymal stem cells (BMSCs) are believed to maintain bone and fat homeostasis by differentiating into osteoblasts and adipocytes. We aimed to explore the role of seipin in the osteogenic/adipogenic differentiation balance of BMSCs. Seipin loxP/loxP mice are used to explore metabolic disorders caused by seipin gene mutations...
February 22, 2024: Stem Cells and Development
https://read.qxmd.com/read/38386505/clinical-evaluation-of-safety-and-efficacy-of-a-central-cgmp-laboratory-produced-autologous-adipose-derived-stromal-vascular-fraction-cell-therapy-product-for-the-treatment-of-knee-osteoarthritis
#13
JOURNAL ARTICLE
Christopher Rogers, Robert J Harman, Mitchell Sheinkop, Peter Hanson, Mary Ambach, Tal David, Rahul Desai, Steve Sampson, Danielle Aufiero, Jay Bowen, Gerard Malanga
Knee osteoarthritis (KOA) is a prevalent condition characterized by the progressive deterioration of the entire joint and has emerged as a prominent contributor to disability on a global scale. The nature of the disease and its impact on joint function significantly limit mobility and daily activities, highlighting its substantial influence on patients' overall well-being. Stromal vascular fraction (SVF) is a heterogenous, autologous cell product, containing mesenchymal stem cells, derived from the patient's subcutaneous adipose tissue with demonstrated safety and efficacy in the treatment of KOA patients...
February 22, 2024: Stem Cells and Development
https://read.qxmd.com/read/38370706/direct-delivery-of-stabilized-cas-embedded-base-editors-achieves-efficient-and-accurate-editing-of-clinically-relevant-targets
#14
Jeong Min Lee, Jing Zeng, Pengpeng Liu, My Anh Nguyen, Diego Suchenski Loustaunau, Daniel E Bauer, Nese Kurt Yilmaz, Scot A Wolfe, Celia A Schiffer
Over the last 5 years, cytosine base editors (CBEs) have emerged as a promising therapeutic tool for specific editing of single nucleotide variants and disrupting specific genes associated with disease. Despite this promise, the currently available CBE's have the significant liabilities of off-target and bystander editing activities, in part due to the mechanism by which they are delivered, causing limitations in their potential applications. In this study we engineeredhighly stabilized Cas-embedded CBEs (sCE_CBEs) that integrate several recent advances, andthat are highly expressible and soluble for direct delivery into cells as ribonucleoprotein (RNP) complexes...
February 8, 2024: bioRxiv
https://read.qxmd.com/read/38366751/nuclear-factor-i-a-nfia-and-nuclear-factor-i-b-nfib-are-jointly-required-for-mouse-postnatal-neural-stem-cell-self-renewal
#15
JOURNAL ARTICLE
Christine E Campbell, Karstin Webber, Jonathan E Bard, Lee D Chaves, Jason M Osinski, Richard M Gronostajski
Mouse postnatal neural stem cells (pNSCs) can be expanded in vitro in the presence of EGF and FGF2 and upon removal of these factors cease proliferation and generate neurons, astrocytes and oligodendrocytes. The genetic requirements for self-renewal and lineage-commitment of pNSCs are incompletely understood. Here we show that the transcription factors NFIA and NFIB, previously shown individually to be essential for the normal commitment of pNSCs to the astrocytic lineage in vivo , are jointly required for normal self-renewal of pNSCs in vitro and in vivo...
February 17, 2024: Stem Cells and Development
https://read.qxmd.com/read/38366745/differentiation-of-human-induced-pluripotent-stem-cell-derived-dental-stem-cells-through-epithelial-mesenchymal-interaction
#16
JOURNAL ARTICLE
Ji-Hye Kim, Jihye Yang, Min-Gi Ki, Dae Hyun Jeon, Jae-Won Kim, Mi Jang, Gene Lee
Research on tooth regeneration using human-induced pluripotent stem cells (hiPSCs) is valuable for autologous dental regeneration. Acquiring mesenchymal and epithelial cells as a resource for dental regeneration is necessary because mesenchymal-epithelial interactions play an essential role in dental development. We reported the establishment of hiPSCs-derived dental epithelial-like cell (EPI-iPSCs), but hiPSCs-derived dental mesenchymal stem cells (MSCs) have not yet been reported. This study was conducted to establish hiPSCs-derived MSCs, and to differentiate them into dental cells with EPI-iPSCs...
February 17, 2024: Stem Cells and Development
https://read.qxmd.com/read/38358434/ovarian-tissue-cryopreservation-for-fertility-preservation-before-hematopoietic-stem-cell-transplantation-in-patients-with-sickle-cell-disease-safety-ovarian-function-follow-up-and-results-of-ovarian-tissue-transplantation
#17
JOURNAL ARTICLE
Mitawa Millin Missontsa, Françoise Bernaudin, Anne Fortin, Nathalie Dhédin, Corinne Pondarré, Karima Yakouben, Bénédicte Neven, Martin Castelle, Marina Cavazzana, Harry Lezeau, Matthieu Peycelon, Annabel Paye-Jaouen, Jeremy Sroussi, Tamara Diesch-Furlanetto, Virginie Barraud-Lange, Sabine Sarnacki, Mony Fahd, Isis Marchand, Clémence Delcour, Dominique Vexiau, Jean-Benoît Arlet, Annie Kamdem, Cécile Arnaud, Jean-Hugues Dalle, Catherine Poirot
PURPOSE: To describe the experience of performing ovarian tissue cryopreservation (OTC) before hematopoietic stem cell transplantation (HSCT), among girls/women with severe sickle cell disease (SCD)(SS or S/β0 -thalassemia) who are, besides the usual surgical risk, at risk of SCD-related complications during the fertility preservation procedure for improving their counseling and management. METHODS: This retrospective study included 75 patients (girls/women) with SCD who have had OTC before myeloablative conditioning regimen (MAC) for HSCT...
February 15, 2024: Journal of Assisted Reproduction and Genetics
https://read.qxmd.com/read/38344818/an-update-review-of-new-therapies-in-sickle-cell-disease-the-prospects-for-drug-combinations
#18
REVIEW
Sanne Lugthart, Catarina Ginete, Patience Kuona, Miguel Brito, Baba Psalm Duniya Inusa
INTRODUCTION: Sickle cell disease (SCD) is an inherited disorder characterised by polymerisation of deoxygenated haemoglobin S and microvascular obstruction. The cardinal feature is generalised pain referred to as vaso-occlusive crises (VOC), multi-organ damage and premature death. SCD is the most prevalent inherited life-threatening disorders in the world and over 85% of world's 400,000 annual births occur low-and-middle-income countries. Hydroxyurea remained the only approved disease modifying therapy (1998) until the FDA approved L-glutamine (2017), Crizanlizumab and Voxelotor (2019) and gene therapies (Exa-cel and Lovo-cel, 2023)...
February 2024: Expert Opinion on Pharmacotherapy
https://read.qxmd.com/read/38326760/the-benefits-of-stem-cell-biology-and-tissue-engineering-in-low-earth-orbit
#19
JOURNAL ARTICLE
Madelyn Arzt, Maedeh Mozneb, Sean Escopete, Jemima Moses, Arun Sharma
Over the past 15 years, there has been a significant shift in biomedical research toward a major focus on stem cell research. Although stem cells and their derivatives exhibit potential in modeling and mitigating human diseases, the ongoing objective is to enhance their utilization and translational potential. Stem cells are increasingly employed in both academic and commercial settings for a variety of in vitro and in vivo applications in regenerative medicine. Notably, accessibility to stem cell research in low-Earth orbit (LEO) has expanded, driven by the unique properties of space, such as microgravity, which cannot exactly be replicated on Earth...
February 26, 2024: Stem Cells and Development
https://read.qxmd.com/read/38326171/role-of-gene-therapy-in-sickle-cell-disease
#20
JOURNAL ARTICLE
Aishwarya Raghuraman, Rebecca Lawrence, Rudrakshi Shetty, Avanthika Chaithanya, Sharan Jhaveri, Brinela Vivas Pichardo, Amulya Mujakari
BACKGROUND: Gene therapy is an emerging treatment for sickle cell disease that works by replacing a defective gene with a healthy gene, allowing the body to produce normal red blood cells. This form of treatment has shown promising results in clinical trials, and is a promising alternative to traditional treatments. Gene therapy involves introducing a healthy gene into the body to replace a defective gene. The new gene can be delivered using a viral vector, which is a modified virus that carries the gene...
February 6, 2024: Disease-a-month: DM
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