New therapy Archives - Page 3 of 15 - Israel Molecular Medicine Portal

Modified Cell Therapy Holds Promise for Rare Autoimmune DisorderSafety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a studyModified Cell Therapy Holds Promise for Rare Autoimmune Disorder

Summary Background Chimeric antigen receptor (CAR) T cells are highly effective in treating haematological malignancies, but associated toxicities and the need for lymphodepletion limit their

Phase 1/2a clinical trial in ALS with ropinirole, a drug candidate identified by iPSC drug discovery

Highlights Summary iPSC-based drug discovery led to a phase 1/2a trial of ropinirole in ALS. 20 participants with sporadic ALS received ropinirole or placebo for

Pembrolizumab in combination with gemcitabine and cisplatin compared with gemcitabine and cisplatin alone for patients with advanced biliary tract cancer (KEYNOTE-966): a randomised, double-blind, placebo-controlled, phase 3 trial

Summary Background Biliary tract cancers, which arise from the intrahepatic or extrahepatic bile ducts and the gallbladder, generally have a poor prognosis and are rising

Disrupting the α-synuclein-ESCRT interaction with a peptide inhibitor mitigates neurodegeneration in preclinical models of Parkinson’s disease

Abstract Accumulation of α-synuclein into toxic oligomers or fibrils is implicated in dopaminergic neurodegeneration in Parkinson’s disease. Here we performed a high-throughput, proteome-wide peptide screen

Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing

Abstract The expanding applications of nonviral genomic medicines in the lung remain restricted by delivery challenges. Here, leveraging a high-throughput platform, we synthesize and screen

T cell immunotherapies engage neutrophils to eliminate tumor antigen escape variants

Highlights Summary Cancer immunotherapies, including adoptive T cell transfer, can be ineffective because tumors evolve to display antigen-loss-variant clones. Therapies that activate multiple branches of the

Trial of Globus Pallidus Focused Ultrasound Ablation in Parkinson’s Disease

Abstract BACKGROUND Unilateral focused ultrasound ablation of the internal segment of globus pallidus has reduced motor symptoms of Parkinson’s disease in open-label studies. METHODS We

Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A

Abstract BACKGROUND Efanesoctocog alfa provides high sustained factor VIII activity by overcoming the von Willebrand factor–imposed half-life ceiling. The efficacy, safety, and pharmacokinetics of efanesoctocog

High-confidence cancer patient stratification through multiomics investigation of DNA repair disorders

Abstract Multiple cancer types have limited targeted therapeutic options, in part due to incomplete understanding of the molecular processes underlying tumorigenesis and significant intra- and

USB1 is a miRNA deadenylase that regulates hematopoietic development

Trimming microRNA for blood development Mutations in the 3′ to 5′ RNA exonuclease USB1 cause a pediatric disease with defects in the production of blood

Stratum Corneum Lipid and Cytokine Biomarkers at Two Months of Age Predict the Future Onset of Atopic Dermatitis

ABSTRACT Background Atopic dermatitis (AD) commonly occurs in children and can progress into severe phenotypes or atopic march, causing significant impairment in quality of life.

Engineering of an electrically charged hydrogel implanted into a traumatic brain injury model for stepwise neuronal tissue reconstruction

Abstract Neural regeneration is extremely difficult to achieve. In traumatic brain injuries, the loss of brain parenchyma volume hinders neural regeneration. In this study, neuronal

Inhibition of cellular RNA methyltransferase abrogates influenza virus capping and replication

Catching out cap snatching Some virus families hijack part of their hosts’ RNA to enable their own replication in a process called cap snatching. Before

A nanofiber-hydrogel composite improves tissue repair in a rat model of Crohn’s disease perianal fistulas

Abstract Perianal fistulas (PAFs) represent a severe complication of Crohn’s disease (CD). Despite the advent of biologic and small-molecule therapeutics for luminal disease, PAFs in

Endothelin blockade prevents the long-term cardiovascular and renal sequelae of acute kidney injury in mice

Abstract Acute kidney injury (AKI) is common and associated with increased risks of cardiovascular and chronic kidney disease. Causative molecular/physiological pathways are poorly defined. There

CAR T therapy extends its reach to autoimmune diseases

CAR T therapy has revolutionized the treatment of hematologic cancers. In their recent Nature Medicine paper, Mackensen et al. report the use of CAR T cells to treat systemic

Targeting Xkr8 via nanoparticle-mediated in situ co-delivery of siRNA and chemotherapy drugs for cancer immunochemotherapy

Abstract Activation of scramblases is one of the mechanisms that regulates the exposure of phosphatidylserine to the cell surface, a process that plays an important

Cytoplasmic pool of U1 spliceosome protein SNRNP70 shapes the axonal transcriptome and regulates motor connectivity

Highlights Summary Regulation of pre-mRNA splicing and polyadenylation plays a profound role in neurons by diversifying the proteome and modulating gene expression in response to

Designed peptides as nanomolar cross-amyloid inhibitors acting via supramolecular nanofiber co-assembly

Abstract Amyloid self-assembly is linked to numerous devastating cell-degenerative diseases. However, designing inhibitors of this pathogenic process remains a major challenge. Cross-interactions between amyloid-β peptide

Epstein-Barr virus gH/gL has multiple sites of vulnerability for virus neutralization and fusion inhibition

Highlights •Six human mAbs target five distinct sites on EBV gH/gL •Some of these antigenic sites match sites of vulnerability on other herpesviruses •Each mAb

Distinct mechanisms of mismatch repair deficiency delineate two modes of response to PD-1 immunotherapy in endometrial carcinoma

Abstract Mismatch repair-deficient (MMRd) cancers have varied responses to immune checkpoint blockade (ICB). We conducted a phase 2 clinical trial of the PD-1 inhibitor pembrolizumab

Brachytherapy via a depot of biopolymer-bound 131I synergizes with nanoparticle paclitaxel in therapy-resistant pancreatic tumours

Abstract Locally advanced pancreatic tumours are highly resistant to conventional radiochemotherapy. Here we show that such resistance can be surmounted by an injectable depot of

A regulatory network of Sox and Six transcription factors initiate a cell fate transformation during hearing regeneration in adult zebrafish

Highlights • Integrated scRNA-seq and scATAC-seq of zebrafish inner ears during hearing regeneration• Sox transcription factors trigger the regeneration response in the support cells• Sox

Discovery of an orally active benzoxaborole prodrug effective in the treatment of Chagas disease in non-human primates

Abstract Trypanosoma cruzi, the agent of Chagas disease, probably infects tens of millions of people, primarily in Latin America, causing morbidity and mortality. The options

In vitro modeling and rescue of ciliopathy associated with IQCB1/NPHP5 mutations using patient-derived cells

Highlights •NPHP5-LCA patient-derived fibroblasts and RPE display abnormally elongated cilia •Outer segment protein localization is impaired in patient-derived photoreceptors •CEP290 protein reduction is observed across

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Category: New therapy