- Adenovirus-Associated Virus Vector-Mediated Gene Transfer in Hemophilia B
[作者：Nathwani, AC; Tuddenham, EGD; Rangarajan, S; Rosales, C; McIntosh, J; Linch, DC; Chowdary, P; Riddell, A; Pie, AJ; Harrington, C; O'Beirne, J; Smith, K; Pasi, J; Glader, B; Rustagi, P; Ng, CYC; Kay, MA; Zhou, JF; Spence, Y; Morton, CL; Allay, J; Coleman, J; Sleep, S; Cunningham, JM; Srivastava, D; Basner-Tschakarjan, E; Mingozzi, F; High, KA; Gray, JT; Reiss, UM; Nienhuis, AW; Davidoff, AM,期刊：New England Journal of Medicine, 页码：2357-2365 , 文章类型： Article，,卷期：2011年365-25]
- BACKGROUND Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder.METHODS We infused a single dose of a serotype-8-pseudotyped,...
- Intensive Diabetes Therapy and Glomerular Filtration Rate in Type 1 Diabetes
[作者：de Boer, IH; Sun, WJ; Cleary, PA; Lachin, JM; Molitch, ME; Steffes, MW; Zinman, B,期刊：New England Journal of Medicine, 页码：2366-2376 , 文章类型： Article，,卷期：2011年365-25]
- BACKGROUND An impaired glomerular filtration rate (GFR) leads to end-stage renal disease and increases the risks of cardiovascular disease and death. Persons with type 1 diabetes are at high risk for kidney disease, but ...
- INF2 Mutations in Charcot-Marie-Tooth Disease with Glomerulopathy
[作者：Boyer, O; Nevo, F; Plaisier, E; Funalot, B; Gribouval, O; Benoit, G; Cong, EH; Arrondel, C; Tete, MJ; Montjean, R; Richard, L; Karras, A; Pouteil-Noble, C; Balafrej, L; Bonnardeaux, A; Canaud, G; Charasse, C; Dantal, J; Deschenes, G; Deteix, P; Dubourg, O; Petiot, P; Pouthier, D; Leguern, E; Guiochon-Mantel, A; Broutin, I; Gubler, MC; Saunier, S; Ronco, P; Vallat, JM; Alonso, MA; Antignac, C; Mollet, G,期刊：New England Journal of Medicine, 页码：2377-2388 , 文章类型： Article，,卷期：2011年365-25]
- BACKGROUND Charcot-Marie-Tooth neuropathy has been reported to be associated with renal diseases, mostly focal segmental glomerulosclerosis (FSGS). However, the common mechanisms underlying the neuropathy and FSGS remain...
- Primary Hyperparathyroidism
[作者：Marcocci, C; Cetani, F,期刊：New England Journal of Medicine, 页码：2389-2397 , 文章类型： Article，,卷期：2011年365-25]
- A 62-year-old woman is found on routine laboratory testing to have a serum calcium level of 10.8 mg per deciliter (2.7 mmol per liter) (normal range, 8.4 to 10.4 [2.1 to 2.7]). The serum intact parathyroid hormone (PTH) ...
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