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Mashup Score: 4
Novel tissue-agnostic therapeutics targeting driver mutations in tumor cells have been recently approved by FDA, driven by basket trials that have demonstrated their efficacy and safety across diverse tumor histology. However, the relative rarity of primary brain tumors (PBTs) has limited their representation in early trials of tissue-agnostic medications. Thus, consensus continues to evolve regarding utility of tissue-agnostic medications in routine practice for PBTs, a diverse group of neoplasms characterized by limited treatment options and unfavorable prognoses. We describe current and potential impact of tissue-agnostic approvals on management of PBTs. We discuss data from clinical trials for PBTs regarding tissue-agnostic targets, including BRAFV600E, neurotrophic tyrosine receptor kinase (NTRK) fusions, microsatellite instability (MSI), mismatch repair deficiency (dMMR), and high tumor mutational burden (TMB-H), in context of challenges in managing PBTs. Described are additional
Source: www.cell.comCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 4Gastruloids generated without exogenous Wnt activation develop anterior neural tissues - 7 hour(s) ago
In this article, Lutolf and colleagues propose a new method to study post-implantation embryonic development in vitro. Aggregates of mouse embryonic stem cells were shown to differentiate into structures with embryo-like patterning and morphogenesis. Furthermore, initial aggregate size and inhibition of Wnt signaling has been shown to be crucial for the axial morphogenesis and emergence of anterior neural progenitors.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 7mRNA vaccine trafficking and resulting protein expression after intramuscular administration - 7 hour(s) ago
Burke and colleagues demonstrated that following intramuscular administration of mRNA-lipid nanoparticles, mRNA was present for 24 h and then rapidly degraded. mRNA and expressed protein were detected in injection site tissue-resident and infiltrating immune cells and draining lymph nodes. mRNA-lipid nanoparticle vaccine trafficking and resulting protein expression are crucial for effective immune response.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 1
A targeted therapy against the kinase MKK4 was developed (HRX215) and investigated preclinically, as well as in a phase I study in humans. HRX215 boosted liver regeneration, prevented liver failure after extensive hepatectomy in pigs, and holds the promise to prevent liver failure after extensive oncological liver resections or transplantation of small liver grafts in humans.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 4Discovery and preclinical development of a therapeutically active nanobody-based chimeric antigen receptor targeting human CD22 - 10 hour(s) ago
McComb and colleagues report on their work to develop a camelid-nanobody-based chimeric antigen receptor-T cell therapy targeting human CD22 antigen. A membrane-proximal domain targeting CD22 nanobody CAR incorporating a CD8-transmembrane domain was selected as lead based on superior in vitro cytotoxicity, specificity, persistence, and in vivo activity.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 1AAV-mediated gene therapy produces fertile offspring in the Lhcgr-deficient mouse model of Leydig cell failure - 11 hour(s) ago
Leydig cell failure results in testosterone deficiency and male infertility. Xia et al. demonstrate AAV-mediated gene therapy recovers testosterone levels, restarts sexual development, restores spermatogenesis, and produces fertile offspring in a mouse model of Leydig cell failure, and thus may have value for future clinical applications.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 1Rescue of Alzheimer’s disease phenotype in a mouse model by transplantation of wild-type hematopoietic stem and progenitor cells - 11 hour(s) ago
Mishra et al. demonstrate that single systemic wild-type hematopoietic stem and progenitor cell transplantation leads to complete preservation of the neurocognitive performance, partial preservation of blood-brain barrier integrity, and reduction of the Aβ plaque, microgliosis, and neuroinflammation in 5xFAD mice. In contrast, 5xFAD HSPC transplantation has no to limited impact.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
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Mashup Score: 2RNA editing: Expanding the potential of RNA therapeutics - 11 hour(s) ago
ADAR-based RNA editing has emerged as a powerful tool to engineer RNAs, enable correction of disease-causing mutations, and modulate protein functions. We review the emerging field of therapeutic RNA editing, highlight recent laboratory advancements, and discuss the key challenges on the path to clinical development.
Source: www.cell.comCategories: General Medicine News, General NewsTweet
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Mashup Score: 5
DrugMap serves as a roadmap to develop covalent ligands for oncogenic drivers.
Source: www.cell.comCategories: General Medicine News, Hem/OncsTweet
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Mashup Score: 1Process intensification for lentiviral vector manufacturing using tangential flow depth filtration - 13 hour(s) ago
Process intensification was achieved using tangential flow depth filtration (TFDF) and stable producer cells to produce an order of magnitude more lentiviral vectors compared with the standard batch process. TFDF membranes demonstrated high filter capacity and efficient separation of lentiviral vectors from producer cells for up to 96 h postinduction.
Source: www.cell.comCategories: General Medicine News, General HCPsTweet
Impact of tissue-agnostic approvals on management of primary brain tumors: Trends in Cancer @trendscancer https://t.co/dJviRX7JoG