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Tag: FAP
Eplontersen may stabilize, slow heart damage in FAP patients
A little more than a year of treatment with eplontersen was found to stabilize or even improve the heart’s structure and function in people with familial amyloid polyneuropathy (FAP) who also were experiencing symptoms of heart disease, known as cardiomyopathy. That’s according to an exploratory analysis of data from the…
Criteria for better assessment of rare genetic variants that can lead to hereditary colorectal cancer
APC gene, APC protein, and criteria boundaries and genotype-phenotype correlations. Representation of the APC gene (bottom) and its main protein product (middle) on the reference sequence NM_000038.6 (non-coding exon 1 not shown). The figure shows on the top the boundaries for the application of PVS1, BS3 and BP1 and genotype-phenotype…
Development of mRNA-Directed Delivery – LNP Delivery System
During the COVID-19 pandemic, the success of mRNA vaccines has greatly propelled the development of mRNA therapeutics. mRNA is a negatively charged nucleic acid that serves as a template for protein synthesis in ribosomes. Despite its utility, the instability of mRNA necessitates appropriate carriers for in vivo delivery. Currently, lipid nanoparticles (LNPs) are…
Insights gained from single-cell analysis of chimeric antigen receptor T-cell immunotherapy in cancer | Military Medical Research
Zhu J, Ke Y, Liu Q, Yang J, Liu F, Xu R, et al. Engineered Lactococcus lactis secreting Flt3L and OX40 ligand for in situ vaccination-based cancer immunotherapy. Nat Commun. 2022;13(1):7466. Article CAS PubMed PubMed Central Google Scholar Evans ER, Bugga P, Asthana V, Drezek R. Metallic nanoparticles for cancer…
Gene editing therapy leads to sustained drop in toxic TTR protein
A single infusion of NTLA-2001, an experimental gene editing therapy, generally is safe and leads to a marked reduction in blood levels of the harmful transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP) and ATTR amyloidosis with cardiomyopathy (ATTR-CM). These updated, interim data cover 65 of 72 patients…
Phase 3 trial of NTLA-2001 for ATTR-CM to launch by year’s end
Intellia Therapeutics is launching a pivotal Phase 3 clinical trial in the U.S. to evaluate the safety and effectiveness of its investigational gene-editing therapy NTLA-2001 in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study, which is expected to start by the end of the year, follows the recent approval…
Radiopharmaceuticals market booms with funding surge
Emerging as a new class of drugs, radiopharmaceuticals are all the rage in cancer treatment research, at present. And now, with American biotech Nucleus RadioPharma raising $56 million to streamline its supply chains and make the drugs more accessible, it seems like the radiopharmaceutical market might be onto something big….
Eplontersen’s efficacy in FAP supported by full Phase 3 trial results
More than a year of treatment with the experimental therapy eplontersen significantly reduces blood levels of transthyretin — the protein that accumulates to toxic levels in familial amyloid polyneuropathy (FAP) — eases nerve damage-related disability, and improves quality of life for patients. That’s according to the full results of the…
Transient naive reprogramming corrects hiPS cells functionally and epigenetically
Cell culture All cell lines used and derived by different approaches in this study are listed in Supplementary Table 1. Detailed information about the experimental design, materials and reagents is presented in the Reporting Summary. Primary human adult dermal fibroblasts (HDFa) from three different female donors were obtained from Gibco…
A multi-database based ceRNA regulatory network for gastric cancer prognosis
Abstract Abstract Objective: Circular RNA(circRNA) is a kind of endogenous non-coding RNA, which may be related to the occurrence and development of cancer. Based on the GEO database, this paper constructs a circRNA as a competitive endogenous RNAs(ceRNAs) that binds with microRNAs (miRNAs) to affect and regulate the expression…
Strategies to therapeutically modulate cytokine action
Cohen, S., Bigazzi, P. E. & Yoshida, T. Commentary. Similarities of T cell function in cell-mediated immunity and antibody production. Cell Immunol. 12, 150–159 (1974). Article CAS PubMed Google Scholar Keegan, A. D. & Leonard, W. J. in Paul’s Fundamental Immunology 8th edn, ch. 9 (eds Flajnik, M. F., Singh,…
Lymph Node Fibroblasts May Suppress T-Cell Function in DLBCL
The immunosuppressive role of fibroblastic reticular cells (FRCs) in diffuse large B-cell lymphoma (DLBCL) is detailed in new research published in The Journal of Clinical Investigation. According to the study’s authors, these findings could have implications for disease pathogenesis and for optimizing immunotherapy in patients with DLBCL. The disease has…
Modelling ligand depletion for simultaneous affinity and binding site quantification on cells and tissue
Cell culture All cells were maintained under standard cell culture conditions (37 °C, 5% CO2, saturated humidity). CHO cells stably transfected with human GIPR (gastric inhibitory polypeptide receptor) were obtained from InSCREENeX GmbH (Germany) and cultured in Dulbecco`s Modified Eagle`s Medium/Nutrient Mixture F-12 Ham (Cat.No. D6421, Sigma) supplemented with 5% fetal bovine…
BMPER is a marker of adipose progenitors and adipocytes and a positive modulator of adipogenesis
Human subject characteristics Visceral (greater omental) adipose tissue samples were obtained in consented patients for our University of Utah Institutional Review Board (IRB)-approved obesity biorepository at the time of laparoscopic bariatric surgery (obesity group) or laparoscopic abdominal surgery for non-cancerous, non-infected general surgery indications (lean group). Patients were fasted for…
Onpattro shows benefits for 1.5 years in ATTR cardiomyopathy trial | Therapy seen to slow down disability in people with ATTR-CM
Treatment with Onpattro (patisiran) for 1.5 years leads to sustained reductions in disability progression among people with ATTR amyloidosis with cardiomyopathy (ATTR-CM), or damage to the heart. That’s according to interim data from the ongoing open-label extension (OLE) portion of the Phase 3 APOLLO-B clinical trial (NCT03997383). That trial’s initial…
Molecular Partners presenting positive solid tumor data
Molecular Partners AG will present additional positive data from the ongoing phase 1 study of MP0317, a CD40 agonist designed to activate immune cells specifically within the tumor microenvironment by anchoring to fibroblast activation protein (FAP), at the 2023 ASCO (American Society of Clinical Oncology) annual meeting, June 2 to…
JCI – Tumor-activated lymph node fibroblasts suppress T cell function in diffuse large B cell lymphoma
Recent transcriptomic-based analysis of diffuse large B cell lymphoma (DLBCL) has highlighted the clinical relevance of lymph node (LN) fibroblast and tumor-infiltrating lymphocyte (TIL) signatures within the tumor microenvironment (TME). However, the immunomodulatory role of fibroblasts in lymphoma remains unclear. Here, by studying human and mouse DLBCL-LNs, we identify the…
KEGG T01001: 11146
Entry 11146 CDS T01001 Symbol GLMN, FAP, FAP48, FAP68, FKBPAP, GLML, GVM, VMGLOM Name (RefSeq) glomulin, FKBP associated protein KO K23345 glomulin Organism hsa Homo sapiens (human) Pathway hsa05131 Shigellosis Network nt06521 NLR signaling Element N00948 Shigella IpaH7.8 to NLRP3 Inflammasome signaling pathway Disease H00531 Venous malformations Brite KEGG Orthology (KO) [BR:hsa00001] 09160 Human Diseases 09171…
AAN 2023: Amvuttra and Onpattro show similar efficacy as FAP treatments | Comparable, sustained drops in transthyretin levels seen
Amvuttra (vutrisiran) and Onpattro (patisiran) showed similar safety and efficacy for familial amyloid polyneuropathy (FAP) patients, according to new analyses from the Phase 3 HELIOS-A trial that supported Amvuttra’s regulatory approval. Both therapies led to similar reductions in transthyretin, the protein that accumulates in FAP, and they were associated with comparable…
Promising Target Antigens for Cell Therapy of Solid Tumors | Lab Equipment
Initially designed for hematological malignancies, CAR-T therapy is now being expanded to target solid tumors using specialized CAR-T cells that attack specific antigens. Ongoing clinical trials are being conducted to evaluate the efficacy of CAR-T cells targeting FAP, GPC3, HER2, L1CAM, CEACAM5, MSLN, IL-13RA2, EpCAM, PSMA, and EGFR. Studies have…
Eplontersen for 1 year found to safely ease symptoms in FAP | All goals met in Phase 3 trial of adults, top-line data show
More than one year of treatment with eplontersen safely reduced symptoms of nerve damage and improved quality of life for adults with familial amyloid polyneuropathy (FAP), according to top-line data from the Phase 3 NEURO-TTRansform trial. The experimental therapy from Ionis Pharmaceuticals and AstraZeneca also significantly lowered blood levels of…
POINT Biopharma Confirms No Disruptions to the Manufacturing and Clinical Supply for the 177Lu-PNT2002 SPLASH Trial, a Phase 3 Study in Patients with Metastatic Castration Resistant Prostate Cancer (mCRPC)
INDIANAPOLIS, March 07, 2023 (GLOBE NEWSWIRE) — POINT Biopharma Global Inc. (NASDAQ: PNT) (the “Company” or “POINT”), a company accelerating the discovery, development, and global access to life-changing radiopharmaceuticals, today affirmed that the SPLASH clinical trial for the PSMA-targeted PNT2002 program is not experiencing any manufacturing or drug supply issues…
Coordinated local RNA overexpression of complement induced by interferon gamma in myositis
Differential expression of complement proteins in the different types of IM To define the transcriptomic profiles of patients with different types of IM, we performed bulk RNAseq on muscle biopsies from 132 IM patients, including those with DM (n = 44), AS (n = 18), IMNM (n = 54), and IBM (n = 16). The DM group included…
Transcriptional reprogramming of skeletal muscle stem cells by the niche environment
Isolation of pure populations of MuSCs and niche cells by fluorescence-activated cell sorting (FACS) Hindlimb muscles were dissected from young (4–6 weeks old) or aged (22–26 months old) C57BL/6J (Jackson Laboratory, 000664) mice and minced until no visible tissue chunks were visible. Muscle was then digested in a 15 ml Falcon…
Single-cell analyses define a continuum of cell state and composition changes in the malignant transformation of polyps to colorectal cancer
Mapping molecular changes across malignant transformation We generated single-cell data for 81 samples collected from eight FAP and seven non-FAP donors (Fig. 1a and Supplementary Tables 1 and 2). For each tissue, we performed matched scATAC-seq and snRNA-seq (10x Genomics). We obtained high-quality single-cell chromatin accessibility profiles for 447,829 cells…