Ask about this productRelated genes to: Klkb1 Blocking Peptide
- Gene:
- KLKB1 NIH gene
- Name:
- kallikrein B1
- Previous symbol:
- KLK3
- Synonyms:
- -
- Chromosome:
- 4q35.2
- Locus Type:
- gene with protein product
- Date approved:
- 1999-04-23
- Date modifiied:
- 2016-10-05
Related products to: Klkb1 Blocking Peptide
Related articles to: Klkb1 Blocking Peptide
- CRISPR-Cas9 has progressed from an experimental tool to a therapeutic modality, marked by the first regulatory approvals of an ex vivo-edited autologous CD34+ hematopoietic stem cell product that induces fetal hemoglobin (CASGEVY/exa-cel). In this narrative review, we synthesize modality-specific molecular diagnostic strategies used across early CRISPR clinical translation. In parallel, early clinical experience has begun to demonstrate the feasibility of in vivo editing, including subretinal delivery for -associated inherited retinal degeneration (EDIT-101 programme) and hepatocyte-targeted lipid nanoparticles (LNPs) for liver-derived targets such as transthyretin and plasma prekallikrein (KLKB1). As translation expands across hematologic, metabolic, ocular and oncology indications, development is increasingly constrained by the predictability and safety of editing outcomes, delivery-determined biodistribution and exposure time, and immune recognition of bacterial Cas9 orthologs and delivery components. We summarize diagnostic readouts for confirming patient genotype, quantifying on-target editing and expression changes, assessing off-target and structural outcomes using orthogonal assays, and monitoring clonal dynamics and immune responses during long-term follow-up. We also discuss how these readouts interface with CMC controls and regulatory expectations for advanced therapy medicinal products (ATMPs), highlighting the need for fit-for-purpose, standardized testing frameworks in early trials. - Source: PubMed
Publication date: 2026/04/02
Rutkowska AdriannaStrózik TadeuszWasiak TomaszCiunowicz DamianKapelan NataliaSzczepaniak NataliaSosnowski JuliuszGoślińska WeronikaBartkowiak JakubBudny-Lewandowska AgataAntończyk PatrycjaMarkiewicz MariaGustaw PiotrFiliks KamilJaskólska MariaStoczyńska-Fidelus Ewelina - Sarcopenia is an age-related condition characterized by progressive muscle mass, strength and physical performance declines, contributing to frailty and adverse health outcomes. Despite increasing interest in molecular biomarkers, longitudinal data with external validation are limited. - Source: PubMed
Kong Sung HyeJeon Ok HeeKim Ji YeonKim MijiKim JinheePark Seung ShinChang Hak ChulWon Chang WonHan Dohyun - Mutations in apolipoprotein L1 (APOL1) provide protection against parasitic infections but increase kidney disease risk in individuals of African ancestry. To better understand APOL1-related pathologies, we examined genetic drivers of circulating APOL1 in individuals of African and European ancestry across multiple cohorts using three proteomic technologies (Olink, SomaLogic, mass spectrometry). Disease-associated G1 and G2 variants were strong -pQTLs for plasma APOL1 measured by Olink and SomaLogic, but not mass spectrometry. Critically, variant effect directions differed between proteomic platforms: positive with Olink and negative with SomaLogic. An additional missense variant (rs2239785), common in Europeans, exhibited the same discrepancy. Furthermore, variants in the kallikrein-kinin system (, , and ) showed strong -pQTL effects via Olink, but not SomaLogic. These results suggest that both intrinsic mutations and extrinsic KKS activity induce conformational changes in the APOL1 protein that are differentially recognized by the proteomic platforms. - Source: PubMed
Publication date: 2026/02/07
Wang Qingbo SHuang JinguoChan Leanne J GHaste NicoleOlsson NiclasGaun AleksandrMcAllister Fiona EMadhireddy DeepthiBaruch AmosCardone Katie MKumar RachitRitchie MarylynSusztak KatalinMelamud EugeneBaryshnikova Anastasia - Inactive ovaries (IO) are a common reproductive disorder in early lactating dairy cows. This condition significantly reduces reproductive efficiency and economic returns. Although many studies have explored the metabolic characteristics of postpartum IO, systematic proteomic analyses using multiple biological samples are still lacking. This study used data-independent acquisition (DIA) proteomics to systematically analyze protein expression profiles in serum, follicular fluid (FF), and ovarian tissues of dairy cows. The goal was to identify potential molecular regulatory mechanisms and key biological pathways involved in IO. Serum, FF, and ovarian tissue samples were collected from six healthy and six IO multiparous Holstein cows at 63 (± 3) days postpartum. These samples were analyzed using DIA-based quantitative proteomics. - Source: PubMed
Publication date: 2026/02/09
Hao YuJiang XuejieBai YunlongXia ChengSong YuxiZheng Jiasan - Hereditary angioedema (HAE) is a rare autosomal dominant disorder marked by episodic, non-urticarial swelling due to C1 esterase inhibitor (C1-INH) deficiency or dysfunction, leading to excessive bradykinin-mediated vascular permeability. While current treatments focus on symptomatic control using C1-INH replacement or kallikrein inhibitors, they require frequent administration and do not address the underlying genetic defect. CRISPR-Cas9 gene-editing technologies, particularly the investigational therapy NTLA-2002, offer a transformative approach by targeting the gene to durably reduce plasma kallikrein levels. This narrative review summarizes the pathophysiology and conventional treatments of HAE, and highlights emerging clinical evidence supporting the safety and efficacy of NTLA-2002. Preliminary trials demonstrate substantial reductions in HAE attack frequency and plasma kallikrein, with minimal adverse events. However, concerns about long-term safety, off-target effects, ethical implications, and accessibility remain. CRISPR-based therapeutics such as NTLA-2002 represent a paradigm shift in the management of HAE and underscore the broader potential of gene editing for genetic disorders. - Source: PubMed
Publication date: 2025/11/07
Jalal LaibaTaimuri Muskan AsimSumbal AnushaIkram AreebaAli TehreemKhan AyeshaAlam SafaArama Umulkhairah OnyioizaYokolo Hermann