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Mashup Score: 18Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery - 1 day(s) ago
By swapping viral fusogenic proteins (which help viruses fuse with and enter host cells) with mammalian skeletal muscle fusogenic proteins, it is possible to create viral vectors specifically targeted to skeletal muscle for gene therapy of Duchenne muscular dystrophy in mouse models.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 75CRISPR-based functional genomics screening in human-pluripotent-stem-cell-derived cell types - 1 day(s) ago
Li et al. review recent advances and existing challenges in the development and application of CRISPR-based functional genomics screening in human-pluripotent-stem-cell-derived cell models. This emerging technology holds great potential for systematically examining gene function in various human cell types and identifying mechanisms and therapeutic targets for human diseases. This field will continue to advance with the rapid development of CRISPR technology and stem cell technology.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 24Activation-neutral gene editing of tonsillar CD4 T cells for functional studies in human ex vivo tonsil cultures - 1 day(s) ago
Morath et al. present a workflow for highly efficient gene editing of tonsil CD4 T cells without disturbing their physiological functions and unique properties. Reintegration of edited and labeled cells in their physiological cell environment enables mechanistic dissection of cell-cell interactions, e.g., in adaptive immunity or during HIV infection.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 70
Kitte and colleagues achieved reprogramming of donor-derived immune cells to find and eliminate diseased cells via mRNA encoding chimeric antigen receptors (CAR). In a head-to-head comparison, they determined that transporting mRNA in lipid nanoparticles is the optimal method for transient generation and functionality of CAR T cells.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 23An autologous ex vivo model for exploring patient-specific responses to viro-immunotherapy in glioblastoma - 1 day(s) ago
Stavrakaki et al. present a co-culture model designed to investigate the interactions between oncolytic virus-infected glioblastoma cells and immune cells derived from the same patient. This model serves as a valuable tool to explore individual patient responses to virus-based immunotherapy.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 87CAR T cell therapy and the tumor microenvironment: Current challenges and opportunities - 1 day(s) ago
The success of chimeric antigen receptor (CAR) T cell therapy has been hindered by barriers intrinsic to the tumor microenvironment, including metabolic fuel competition, physical barriers to effective CAR T cell infiltration, and immunosuppressive cytokines and cell types. We review novel strategies and sophisticated CAR designs that may overcome these barriers.
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Mashup Score: 31Gain-of-function MYCN causes a megalencephaly-polydactyly syndrome manifesting mirror phenotypes of Feingold syndrome - 1 day(s) ago
Unveiling gain-of-function MYCN role through exploring two megalencephalic patients with polydactyly and gain-of-function MYCN variants, p.Thr58Met and p.Pro60Leu. The mouse model recapitulates the human phenotypes, megalencephaly and polydactyly, mirroring Feingold syndrome. MYCN’s proliferative role extends beyond brain development, impacting kidney and female reproductive tract development, illuminating new disease insights.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 36Empowering pancreatic tumor homing with augmented anti-tumor potency of CXCR2-tethered CAR-NK cells - 1 day(s) ago
Jang and colleagues found that extensive profiling of pancreatic cancer chemokines enabled the genetic enhancement of anti-MSLN CAR-pNK cells with CXCR1 or CXCR2. CXCR2-tethered CAR-NK cells improved tumor-homing, showing synergistic anti-tumor activity in patient-derived organoids for more effective pancreatic cancer treatment.
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Mashup Score: 5
Leone, Gelati, and colleagues assessed the intracerebroventricular injection (ICVI) of allogeneic human neural stem/progenitor cells (hNSCs) for the treatment of progressive multiple sclerosis. The ICVI of highly purified and stable hNSC lines displays an optimal feasibility, safety, and tolerability profile.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 15Utilizing directed evolution to interrogate and optimize CRISPR/Cas guide RNA scaffolds - 1 day(s) ago
Bush et al. present a high-throughput selection and RNA evolution scheme that can identify functional sgRNA scaffold domains with distinct properties. These variants provide additional insight into the structure-activity-relationships required for effective Cas9 guided DNA editing. BLADE SELEX promises to facilitate the development of sgRNA variants with new activities.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
Enveloped viruses pseudotyped with mammalian myogenic cell fusogens target skeletal muscle for gene delivery - read more from @CincyChildrens @doug_millay & colleagues in @CellCellPress #ASGCT2024 https://t.co/bwnm7wE0nH