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ADPKDマウスモデルにおける嚢胞指数の応用
The kidneys of autosomal dominant polycystic kidney disease (ADPKD) patients progressively enlarge due to the formation and expansion of fluid-filled cysts. However, currently there is no available therapy for ADPKD patients that targets the growth of cysts,...
前臨床研究において腎機能のトランスレーショナルな測定値を組み込むことの重要性：経皮的GFR測定
Preclinical Nephrology Research Preclinical studies aiming to assess kidney function in animal models of kidney disease or in response to a test compound have traditionally included readouts such as serum creatinine and blood urea nitrogen (BUN). However, the use of...
白質萎縮におけるeIF2Bモジュレーター
Vanishing White Matter (VWM) disease is a rare and fatal leukodystrophy, caused by recessive mutations in the five genes encoding the protein complex eukaryotic translation initiation factor 2B (eIF2B). eIF2B plays a crucial role in initiating and regulating the...
腎臓疾患の研究モデルが臨床応用に関連していることを、どのように確認すればよいでしょうか？
Although multiple kidney disease models have been described in literature, not all of the models are well-characterized and suitable for preclinical research. To help accelerate the development of new drugs to improve the quality of life of patients living with a...
腹膜癌モデルにおける有効性評価のための腹膜癌指標
Peritoneal carcinomatosis (PC) represents an extreme form of metastatic cancer progression, commonly occurring in gastric, ovarian and colorectal cancers. Metastatic tumor models leading to PC help better understand the toxicities, timing and efficacy of novel...
Stxbp1欠損/存在てんかん性脳症モデルにおける新規抗てんかん薬の縦断的有効性評価
Previously, we have briefly introduced you to the Stxbp1 +/- mouse model of early infantile epileptic encephalopathies. Patients with a mutation in the STXBP1 gene (Syntaxin-binding protein 1, encoding the presynaptic protein Munc18-1) can receive a variety of...
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