Anti-Mouse UNC13D (FLJ0067), Rabbit Polyclonal
- Known as:
- Antibody toMouse UNC13D (FLJ0067), Rabbit Polyclonal
- Catalog number:
- mfl0067
- Product Quantity:
- EUR
- Category:
- -
- Supplier:
- Sceti
- Gene target:
- Anti-Mouse UNC13D (FLJ0067) Rabbit Polyclonal
Related genes to: Anti-Mouse UNC13D (FLJ0067), Rabbit Polyclonal
- Gene:
- UNC13D NIH gene
- Name:
- unc-13 homolog D
- Previous symbol:
- -
- Synonyms:
- Munc13-4
- Chromosome:
- 17q25.3
- Locus Type:
- gene with protein product
- Date approved:
- 2003-10-16
- Date modifiied:
- 2019-04-23
Related products to: Anti-Mouse UNC13D (FLJ0067), Rabbit Polyclonal
Related articles to: Anti-Mouse UNC13D (FLJ0067), Rabbit Polyclonal
- Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening hyperinflammatory syndrome caused by genetic defects in cytotoxic lymphocyte function. Current therapies can control disease activity, but transplantation of allogeneic hematopoietic stem and progenitor cells (HSPCs) remains the only curative option and is associated with substantial risks. These limitations have accelerated development of genome editing approaches enabling precise correction of disease-causing mutations in autologous cells. Familial HLH (FHL) represents a compelling target for genome editing, but successful and safe clinical translation has remained challenging. Preclinical studies demonstrate that targeted editing of key genes, such as and , can restore cytotoxic function in HSPCs and T cells. Translation to the clinic, however, depends on multiple factors, including the choice of target cell population, the level of functional correction required, and gene-specific constraints such as locus complexity and regulation of gene expression. In this review, we synthesize current progress in genome editing for FHL and highlight critical biological and technical barriers to clinical implementation. We propose a conceptual framework for designing genome editing strategies tailored to FHL, emphasizing the alignment of editing platform, gene architecture, and cellular context to enable effective and clinically translatable therapies. - Source: PubMed
Publication date: 2026/07/02
Minaiyan GhazaleAussel ClotildeAmmann SandraCathomen Toni - This retrospective study aimed to investigate the clinical characteristics, management, and prognosis of pediatric malignancy-associated hemophagocytic lymphohistiocytosis (M-HLH). - Source: PubMed
Publication date: 2026/05/07
Huang ChengshuangHuang JiaLe QuZhou Yan - Hemophagocytic lymphohistiocytosis (HLH) is a hyperinflammatory syndrome frequently involving the central nervous system (CNS) and associated with poor outcomes. Neurological manifestations may precede or obscure systemic features, resulting in diagnostic challenges. - Source: PubMed
Publication date: 2026/04/30
Jang SeoyunKim Hye JinCha Jong HoKim JiminYoon HeejeongKim WoojoongChoi Jung YoonKang Hyoung JinKim Man JinKim Soo YeonChae Jong-Hee - Base editors enable precise correction of point mutations without requiring DNA double-strand breaks, yet platform- and cell type-specific genotoxicities remain incompletely characterized. Here, we applied cytosine base editing (CBE) to disrupt a cryptic splice-site mutation in the Unc13d locus of Jinx mice, a model of familial hemophagocytic lymphohistiocytosis type 3 (FHL3). Efficient editing (62%-89%) in fibroblasts, T cells, and hematopoietic stem cells (HSCs) restored Unc13d splicing, reconstituted cytotoxic T cell function, and protected mice from virus-triggered hyperinflammation after transplantation of edited HSCs. Comparative genotoxicity profiling revealed distinct platform- and cell type-specific patterns: hyperactive CBE induced broader off-target activity and more structural variants than CRISPR-Cas9. Although off-target sequence edits persisted, the stability of CBE-induced chromosomal translocations differed between cell types. These findings establish base editing as a therapeutic strategy for a genetically predisposed hyperinflammatory syndrome and underscore the importance of context-specific safety profiling to guide the clinical translation of genome editors. - Source: PubMed
Publication date: 2026/05/13
Lei LeiKaufmann Masako MLao JessicaThoulass GudrunAmmann SandraXiao HuiRhiel ManuelDettmer-Monaco VivianeGrünewald JulianAndrieux GeoffroyAlzubi JamalMiller Bret RWeißert KristofferGräßel LindaSchell ChristophIllert Anna LJoung J KeithBoerries MelanieCornu Tatjana IEhl StephanErlacher MiriamAichele PeterCathomen Toni - To investigate the clinical and laboratory features, phenotypic characteristics, associated genes, and treatment strategies for adult primary hemophagocytic lymphohistiocytosis (HLH). - Source: PubMed
Publication date: 2026/04/17
Huang LeiGuo Shuli